                         SEQUENCE LISTING

<110>  The Trustees of the University of Pennsylvania
 
<120>  COMPOSITIONS AND USES THEREOF FOR TREATMENT OF ANGELMAN SYNDROME

<130>  UPN-21-9579.PCT

<150>  US 63/119,860
<151>  2020-12-01

<150>  US 63/179,807
<151>  2021-04-26

<160>  25    

<170>  PatentIn version 3.5

<210>  1
<211>  3804
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector genome hSyn.hUbe3a-1.GSco.4XmiRNA183.SV40


<220>
<221>  repeat_region
<222>  (1)..(130)
<223>  ITR

<220>
<221>  promoter
<222>  (213)..(678)
<223>  hSynapsin

<220>
<221>  misc_feature
<222>  (690)..(695)
<223>  Kozak

<220>
<221>  CDS
<222>  (696)..(3257)
<223>  hUBE3a-1

<220>
<221>  misc_feature
<222>  (779)..(779)
<223>  mut

<220>
<221>  misc_feature
<222>  (824)..(824)
<223>  mut

<220>
<221>  misc_feature
<222>  (1028)..(1028)
<223>  mut

<220>
<221>  misc_feature
<222>  (1128)..(1128)
<223>  mut

<220>
<221>  misc_feature
<222>  (1181)..(1181)
<223>  mut

<220>
<221>  misc_feature
<222>  (1217)..(1217)
<223>  mut

<220>
<221>  misc_feature
<222>  (1283)..(1283)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (1715)..(1715)
<223>  mut

<220>
<221>  misc_feature
<222>  (1721)..(1721)
<223>  mut

<220>
<221>  misc_feature
<222>  (1766)..(1766)
<223>  mut

<220>
<221>  misc_feature
<222>  (1805)..(1805)
<223>  mut

<220>
<221>  misc_feature
<222>  (1814)..(1814)
<223>  mut

<220>
<221>  misc_feature
<222>  (1991)..(1991)
<223>  mut

<220>
<221>  misc_feature
<222>  (2063)..(2063)
<223>  mut

<220>
<221>  misc_feature
<222>  (2111)..(2111)
<223>  mut

<220>
<221>  misc_feature
<222>  (2294)..(2294)
<223>  mut

<220>
<221>  misc_feature
<222>  (2439)..(2440)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (2723)..(2723)
<223>  mut

<220>
<221>  misc_feature
<222>  (2957)..(2957)
<223>  mut

<220>
<221>  misc_feature
<222>  (3023)..(3023)
<223>  mut

<220>
<221>  misc_feature
<222>  (3056)..(3056)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (3152)..(3152)
<223>  mut

<220>
<221>  misc_feature
<222>  (3161)..(3161)
<223>  mut

<220>
<221>  misc_feature
<222>  (3230)..(3230)
<223>  mut

<220>
<221>  misc_feature
<222>  (3264)..(3285)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3318)..(3311)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3318)..(3339)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3346)..(3367)
<223>  miRNA183

<220>
<221>  polyA_signal
<222>  (3379)..(3610)
<223>  SV40 polyA

<220>
<221>  repeat_region
<222>  (3675)..(3804)
<223>  ITR

<400>  1
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct tgtagttaat gattaacccg ccatgctact tatctacgta gccatgctct      180

aggaagatcc tctagaacta tagctagcat gcctgcagag ggccctgcgt atgagtgcaa      240

gtgggtttta ggaccaggat gaggcggggt gggggtgcct acctgacgac cgaccccgac      300

ccactggaca agcacccaac ccccattccc caaattgcgc atcccctatc agagaggggg      360

aggggaaaca ggatgcggcg aggcgcgtgc gcactgccag cttcagcacc gcggacagtg      420

ccttcgcccc cgcctggcgg cgcgcgccac cgccgcctca gcactgaagg cgcgctgacg      480

tcactcgccg gtcccccgca aactcccctt cccggccacc ttggtcgcgt ccgcgccgcc      540

gccggcccag ccggaccgca ccacgcgagg cgcgagatag gggggcacgg gcgcgaccat      600

ctgcgctgcg gcgccggcga ctcagcgctg cctcagtctg cggtgggcag cggaggagtc      660

gtgtcgtgcc tgagagcgca gtcgaattcg ccacc atg aag agg gca gca gca         713
                                       Met Lys Arg Ala Ala Ala            
                                       1               5                  

aag cac ctg atc gag aga tac tat cac cag ctg acc gag gga tgc gga        761
Lys His Leu Ile Glu Arg Tyr Tyr His Gln Leu Thr Glu Gly Cys Gly           
            10                  15                  20                    

aac gag gca tgt aca aac gag ttc tgc gcc tcc tgt ccc acc ttt ctg        809
Asn Glu Ala Cys Thr Asn Glu Phe Cys Ala Ser Cys Pro Thr Phe Leu           
        25                  30                  35                        

agg atg gat aac aac gcc gcc gcc atc aag gcc ctg gag ctg tac aag        857
Arg Met Asp Asn Asn Ala Ala Ala Ile Lys Ala Leu Glu Leu Tyr Lys           
    40                  45                  50                            

atc aac gcc aag ctg tgc gac ccc cac cct agc aag aag ggc gcc agc        905
Ile Asn Ala Lys Leu Cys Asp Pro His Pro Ser Lys Lys Gly Ala Ser           
55                  60                  65                  70            

tcc gcc tat ctg gag aac tcc aag ggc gcc cct aac aat agc tgt tcc        953
Ser Ala Tyr Leu Glu Asn Ser Lys Gly Ala Pro Asn Asn Ser Cys Ser           
                75                  80                  85                

gag atc aag atg aat aag aag ggc gcc cgg atc gat ttc aag gac gtg       1001
Glu Ile Lys Met Asn Lys Lys Gly Ala Arg Ile Asp Phe Lys Asp Val           
            90                  95                  100                   

acc tac ctg aca gag gag aag gtg tac gag atc ctg gag ctg tgc cgg       1049
Thr Tyr Leu Thr Glu Glu Lys Val Tyr Glu Ile Leu Glu Leu Cys Arg           
        105                 110                 115                       

gag aga gag gat tac agc cca ctg atc aga gtg atc ggc aga gtg ttc       1097
Glu Arg Glu Asp Tyr Ser Pro Leu Ile Arg Val Ile Gly Arg Val Phe           
    120                 125                 130                           

tct agc gcc gag gcc ctg gtg cag tcc ttt aga aag gtg aag cag cac       1145
Ser Ser Ala Glu Ala Leu Val Gln Ser Phe Arg Lys Val Lys Gln His           
135                 140                 145                 150           

aca aag gag gag ctg aag tct ctg cag gcc aag gac gag gac aag gac       1193
Thr Lys Glu Glu Leu Lys Ser Leu Gln Ala Lys Asp Glu Asp Lys Asp           
                155                 160                 165               

gag gac gag aag gag aag gca gcc tgt tct gcc gca gca atg gag gag       1241
Glu Asp Glu Lys Glu Lys Ala Ala Cys Ser Ala Ala Ala Met Glu Glu           
            170                 175                 180                   

gac agc gag gca tcc tct agc cgg atc ggc gat tcc tct caa ggc gac       1289
Asp Ser Glu Ala Ser Ser Ser Arg Ile Gly Asp Ser Ser Gln Gly Asp           
        185                 190                 195                       

aac aat ctg cag aag ctg ggc ccc gac gac gtg agc gtg gat atc gac       1337
Asn Asn Leu Gln Lys Leu Gly Pro Asp Asp Val Ser Val Asp Ile Asp           
    200                 205                 210                           

gcc atc cgg aga gtg tac aca aga ctg ctg agc aac gag aag atc gag       1385
Ala Ile Arg Arg Val Tyr Thr Arg Leu Leu Ser Asn Glu Lys Ile Glu           
215                 220                 225                 230           

acc gcc ttc ctg aac gcc ctg gtg tat ctg agc cct aat gtg gag tgc       1433
Thr Ala Phe Leu Asn Ala Leu Val Tyr Leu Ser Pro Asn Val Glu Cys           
                235                 240                 245               

gat ctg acc tac cac aac gtg tac tcc cgg gac cca aac tac ctg aat       1481
Asp Leu Thr Tyr His Asn Val Tyr Ser Arg Asp Pro Asn Tyr Leu Asn           
            250                 255                 260                   

ctg ttc atc atc gtg atg gag aac aga aat ctg cac tcc ccc gag tat       1529
Leu Phe Ile Ile Val Met Glu Asn Arg Asn Leu His Ser Pro Glu Tyr           
        265                 270                 275                       

ctg gag atg gcc ctg cct ctg ttt tgt aag gcc atg tcc aag ctg cct       1577
Leu Glu Met Ala Leu Pro Leu Phe Cys Lys Ala Met Ser Lys Leu Pro           
    280                 285                 290                           

ctg gca gca cag ggc aag ctg atc agg ctg tgg tct aag tac aac gcc       1625
Leu Ala Ala Gln Gly Lys Leu Ile Arg Leu Trp Ser Lys Tyr Asn Ala           
295                 300                 305                 310           

gat cag atc agg cgc atg atg gag acc ttc cag cag ctg atc aca tac       1673
Asp Gln Ile Arg Arg Met Met Glu Thr Phe Gln Gln Leu Ile Thr Tyr           
                315                 320                 325               

aaa gtg atc tct aac gag ttt aat agc cgc aac ctg gtg aac gac gac       1721
Lys Val Ile Ser Asn Glu Phe Asn Ser Arg Asn Leu Val Asn Asp Asp           
            330                 335                 340                   

gac gcc atc gtg gcc gcc tct aag tgc ctg aag atg gtg tac tac gcc       1769
Asp Ala Ile Val Ala Ala Ser Lys Cys Leu Lys Met Val Tyr Tyr Ala           
        345                 350                 355                       

aac gtg gtg ggc ggc gag gtg gac aca aac cac aac gag gag gac gac       1817
Asn Val Val Gly Gly Glu Val Asp Thr Asn His Asn Glu Glu Asp Asp           
    360                 365                 370                           

gag gag cca atc ccc gag agc tcc gag ctg acc ctg cag gag ctg ctg       1865
Glu Glu Pro Ile Pro Glu Ser Ser Glu Leu Thr Leu Gln Glu Leu Leu           
375                 380                 385                 390           

gga gag gag cgg aga aat aag aag gga cca agg gtg gat cct ctg gag       1913
Gly Glu Glu Arg Arg Asn Lys Lys Gly Pro Arg Val Asp Pro Leu Glu           
                395                 400                 405               

acc gag ctg ggc gtg aag aca ctg gac tgc aga aag cct ctg atc cca       1961
Thr Glu Leu Gly Val Lys Thr Leu Asp Cys Arg Lys Pro Leu Ile Pro           
            410                 415                 420                   

ttc gag gag ttt atc aac gag ccc ctg aac gag gtg ctg gag atg gat       2009
Phe Glu Glu Phe Ile Asn Glu Pro Leu Asn Glu Val Leu Glu Met Asp           
        425                 430                 435                       

aag gac tac acc ttc ttt aag gtg gag aca gag aac aag ttc agc ttt       2057
Lys Asp Tyr Thr Phe Phe Lys Val Glu Thr Glu Asn Lys Phe Ser Phe           
    440                 445                 450                           

atg acc tgt cct ttc atc ctg aac gcc gtg acc aag aat ctg ggc ctg       2105
Met Thr Cys Pro Phe Ile Leu Asn Ala Val Thr Lys Asn Leu Gly Leu           
455                 460                 465                 470           

tac tac gat aac agg atc cgc atg tac tcc gag agg cgc atc acc gtg       2153
Tyr Tyr Asp Asn Arg Ile Arg Met Tyr Ser Glu Arg Arg Ile Thr Val           
                475                 480                 485               

ctg tat tct ctg gtg cag ggc cag cag ctg aat cct tac ctg agg ctg       2201
Leu Tyr Ser Leu Val Gln Gly Gln Gln Leu Asn Pro Tyr Leu Arg Leu           
            490                 495                 500                   

aag gtg cgg aga gac cac atc atc gat gac gcc ctg gtg cgc ctg gag       2249
Lys Val Arg Arg Asp His Ile Ile Asp Asp Ala Leu Val Arg Leu Glu           
        505                 510                 515                       

atg atc gcc atg gag aat cca gcc gat ctg aag aag cag ctg tac gtg       2297
Met Ile Ala Met Glu Asn Pro Ala Asp Leu Lys Lys Gln Leu Tyr Val           
    520                 525                 530                           

gag ttt gag gga gag cag gga gtg gac gag gga ggc gtg tct aag gag       2345
Glu Phe Glu Gly Glu Gln Gly Val Asp Glu Gly Gly Val Ser Lys Glu           
535                 540                 545                 550           

ttc ttt cag ctg gtg gtg gag gag atc ttc aac ccc gat atc ggc atg       2393
Phe Phe Gln Leu Val Val Glu Glu Ile Phe Asn Pro Asp Ile Gly Met           
                555                 560                 565               

ttt acc tac gac gag agc aca aag ctg ttc tgg ttt aat cct tct tcc       2441
Phe Thr Tyr Asp Glu Ser Thr Lys Leu Phe Trp Phe Asn Pro Ser Ser           
            570                 575                 580                   

ttc gag acc gag ggc cag ttt aca ctg atc ggc atc gtg ctg ggc ctg       2489
Phe Glu Thr Glu Gly Gln Phe Thr Leu Ile Gly Ile Val Leu Gly Leu           
        585                 590                 595                       

gcc atc tac aac aat tgt atc ctg gac gtg cac ttc cca atg gtg gtg       2537
Ala Ile Tyr Asn Asn Cys Ile Leu Asp Val His Phe Pro Met Val Val           
    600                 605                 610                           

tat agg aag ctg atg ggc aag aag ggc acc ttt cgc gat ctg ggc gac       2585
Tyr Arg Lys Leu Met Gly Lys Lys Gly Thr Phe Arg Asp Leu Gly Asp           
615                 620                 625                 630           

tcc cac ccc gtg ctg tac cag tct ctg aag gat ctg ctg gag tat gag       2633
Ser His Pro Val Leu Tyr Gln Ser Leu Lys Asp Leu Leu Glu Tyr Glu           
                635                 640                 645               

ggc aac gtg gag gat gac atg atg atc acc ttc cag atc tcc cag aca       2681
Gly Asn Val Glu Asp Asp Met Met Ile Thr Phe Gln Ile Ser Gln Thr           
            650                 655                 660                   

gac ctg ttt ggc aac cca atg atg tac gat ctg aag gag aac ggc gac       2729
Asp Leu Phe Gly Asn Pro Met Met Tyr Asp Leu Lys Glu Asn Gly Asp           
        665                 670                 675                       

aag atc ccc atc aca aac gag aat aga aag gag ttc gtg aac ctg tac       2777
Lys Ile Pro Ile Thr Asn Glu Asn Arg Lys Glu Phe Val Asn Leu Tyr           
    680                 685                 690                           

agc gat tat atc ctg aat aag tcc gtg gag aag cag ttc aag gcc ttt       2825
Ser Asp Tyr Ile Leu Asn Lys Ser Val Glu Lys Gln Phe Lys Ala Phe           
695                 700                 705                 710           

agg cgc ggc ttc cac atg gtg acc aac gag agc cct ctg aag tat ctg       2873
Arg Arg Gly Phe His Met Val Thr Asn Glu Ser Pro Leu Lys Tyr Leu           
                715                 720                 725               

ttt agg cca gag gag atc gag ctg ctg atc tgc ggc tcc cgc aat ctg       2921
Phe Arg Pro Glu Glu Ile Glu Leu Leu Ile Cys Gly Ser Arg Asn Leu           
            730                 735                 740                   

gac ttt cag gcc ctg gag gag acc aca gag tac gac ggc ggc tat acc       2969
Asp Phe Gln Ala Leu Glu Glu Thr Thr Glu Tyr Asp Gly Gly Tyr Thr           
        745                 750                 755                       

agg gac tct gtg ctg atc cgc gag ttc tgg gag atc gtg cac agc ttt       3017
Arg Asp Ser Val Leu Ile Arg Glu Phe Trp Glu Ile Val His Ser Phe           
    760                 765                 770                           

aca gac gag cag aag cgg ctg ttc ctg cag ttt acc acc ggc acc gac       3065
Thr Asp Glu Gln Lys Arg Leu Phe Leu Gln Phe Thr Thr Gly Thr Asp           
775                 780                 785                 790           

aga gca cca gtg gga gga ctg ggc aag ctg aag atg atc atc gcc aag       3113
Arg Ala Pro Val Gly Gly Leu Gly Lys Leu Lys Met Ile Ile Ala Lys           
                795                 800                 805               

aac ggc cca gac aca gag agg ctg ccc acc agc cac acc tgt ttc aac       3161
Asn Gly Pro Asp Thr Glu Arg Leu Pro Thr Ser His Thr Cys Phe Asn           
            810                 815                 820                   

gtg ctg ctg ctg ccc gag tac tcc tct aag gag aag ctg aag gag cgc       3209
Val Leu Leu Leu Pro Glu Tyr Ser Ser Lys Glu Lys Leu Lys Glu Arg           
        825                 830                 835                       

ctg ctg aag gcc atc acc tac gcc aag ggc ttt ggc atg ctg tga tga       3257
Leu Leu Lys Ala Ile Thr Tyr Ala Lys Gly Phe Gly Met Leu                   
    840                 845                 850                           

ggtaccagtg aattctacca gtgccatagg atagtgaatt ctaccagtgc catacacgtg     3317

agtgaattct accagtgcca tagcatgcag tgaattctac cagtgccata ggcggccgct     3377

tcgagcagac atgataagat acattgatga gtttggacaa accacaacta gaatgcagtg     3437

aaaaaaatgc tttatttgtg aaatttgtga tgctattgct ttatttgtaa ccattataag     3497

ctgcaataaa caagttaaca acaacaattg cattcatttt atgtttcagg ttcaggggga     3557

gatgtgggag gttttttaaa gcaagtaaaa cctctacaaa tgtggtaaaa tcgataagga     3617

tcttcctaga gcatggctac gtagataagt agcatggcgg gttaatcatt aactacaagg     3677

aacccctagt gatggagttg gccactccct ctctgcgcgc tcgctcgctc actgaggccg     3737

ggcgaccaaa ggtcgcccga cgcccgggct ttgcccgggc ggcctcagtg agcgagcgag     3797

cgcgcag                                                               3804


<210>  2
<211>  852
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Synthetic Construct

<400>  2

Met Lys Arg Ala Ala Ala Lys His Leu Ile Glu Arg Tyr Tyr His Gln 
1               5                   10                  15      


Leu Thr Glu Gly Cys Gly Asn Glu Ala Cys Thr Asn Glu Phe Cys Ala 
            20                  25                  30          


Ser Cys Pro Thr Phe Leu Arg Met Asp Asn Asn Ala Ala Ala Ile Lys 
        35                  40                  45              


Ala Leu Glu Leu Tyr Lys Ile Asn Ala Lys Leu Cys Asp Pro His Pro 
    50                  55                  60                  


Ser Lys Lys Gly Ala Ser Ser Ala Tyr Leu Glu Asn Ser Lys Gly Ala 
65                  70                  75                  80  


Pro Asn Asn Ser Cys Ser Glu Ile Lys Met Asn Lys Lys Gly Ala Arg 
                85                  90                  95      


Ile Asp Phe Lys Asp Val Thr Tyr Leu Thr Glu Glu Lys Val Tyr Glu 
            100                 105                 110         


Ile Leu Glu Leu Cys Arg Glu Arg Glu Asp Tyr Ser Pro Leu Ile Arg 
        115                 120                 125             


Val Ile Gly Arg Val Phe Ser Ser Ala Glu Ala Leu Val Gln Ser Phe 
    130                 135                 140                 


Arg Lys Val Lys Gln His Thr Lys Glu Glu Leu Lys Ser Leu Gln Ala 
145                 150                 155                 160 


Lys Asp Glu Asp Lys Asp Glu Asp Glu Lys Glu Lys Ala Ala Cys Ser 
                165                 170                 175     


Ala Ala Ala Met Glu Glu Asp Ser Glu Ala Ser Ser Ser Arg Ile Gly 
            180                 185                 190         


Asp Ser Ser Gln Gly Asp Asn Asn Leu Gln Lys Leu Gly Pro Asp Asp 
        195                 200                 205             


Val Ser Val Asp Ile Asp Ala Ile Arg Arg Val Tyr Thr Arg Leu Leu 
    210                 215                 220                 


Ser Asn Glu Lys Ile Glu Thr Ala Phe Leu Asn Ala Leu Val Tyr Leu 
225                 230                 235                 240 


Ser Pro Asn Val Glu Cys Asp Leu Thr Tyr His Asn Val Tyr Ser Arg 
                245                 250                 255     


Asp Pro Asn Tyr Leu Asn Leu Phe Ile Ile Val Met Glu Asn Arg Asn 
            260                 265                 270         


Leu His Ser Pro Glu Tyr Leu Glu Met Ala Leu Pro Leu Phe Cys Lys 
        275                 280                 285             


Ala Met Ser Lys Leu Pro Leu Ala Ala Gln Gly Lys Leu Ile Arg Leu 
    290                 295                 300                 


Trp Ser Lys Tyr Asn Ala Asp Gln Ile Arg Arg Met Met Glu Thr Phe 
305                 310                 315                 320 


Gln Gln Leu Ile Thr Tyr Lys Val Ile Ser Asn Glu Phe Asn Ser Arg 
                325                 330                 335     


Asn Leu Val Asn Asp Asp Asp Ala Ile Val Ala Ala Ser Lys Cys Leu 
            340                 345                 350         


Lys Met Val Tyr Tyr Ala Asn Val Val Gly Gly Glu Val Asp Thr Asn 
        355                 360                 365             


His Asn Glu Glu Asp Asp Glu Glu Pro Ile Pro Glu Ser Ser Glu Leu 
    370                 375                 380                 


Thr Leu Gln Glu Leu Leu Gly Glu Glu Arg Arg Asn Lys Lys Gly Pro 
385                 390                 395                 400 


Arg Val Asp Pro Leu Glu Thr Glu Leu Gly Val Lys Thr Leu Asp Cys 
                405                 410                 415     


Arg Lys Pro Leu Ile Pro Phe Glu Glu Phe Ile Asn Glu Pro Leu Asn 
            420                 425                 430         


Glu Val Leu Glu Met Asp Lys Asp Tyr Thr Phe Phe Lys Val Glu Thr 
        435                 440                 445             


Glu Asn Lys Phe Ser Phe Met Thr Cys Pro Phe Ile Leu Asn Ala Val 
    450                 455                 460                 


Thr Lys Asn Leu Gly Leu Tyr Tyr Asp Asn Arg Ile Arg Met Tyr Ser 
465                 470                 475                 480 


Glu Arg Arg Ile Thr Val Leu Tyr Ser Leu Val Gln Gly Gln Gln Leu 
                485                 490                 495     


Asn Pro Tyr Leu Arg Leu Lys Val Arg Arg Asp His Ile Ile Asp Asp 
            500                 505                 510         


Ala Leu Val Arg Leu Glu Met Ile Ala Met Glu Asn Pro Ala Asp Leu 
        515                 520                 525             


Lys Lys Gln Leu Tyr Val Glu Phe Glu Gly Glu Gln Gly Val Asp Glu 
    530                 535                 540                 


Gly Gly Val Ser Lys Glu Phe Phe Gln Leu Val Val Glu Glu Ile Phe 
545                 550                 555                 560 


Asn Pro Asp Ile Gly Met Phe Thr Tyr Asp Glu Ser Thr Lys Leu Phe 
                565                 570                 575     


Trp Phe Asn Pro Ser Ser Phe Glu Thr Glu Gly Gln Phe Thr Leu Ile 
            580                 585                 590         


Gly Ile Val Leu Gly Leu Ala Ile Tyr Asn Asn Cys Ile Leu Asp Val 
        595                 600                 605             


His Phe Pro Met Val Val Tyr Arg Lys Leu Met Gly Lys Lys Gly Thr 
    610                 615                 620                 


Phe Arg Asp Leu Gly Asp Ser His Pro Val Leu Tyr Gln Ser Leu Lys 
625                 630                 635                 640 


Asp Leu Leu Glu Tyr Glu Gly Asn Val Glu Asp Asp Met Met Ile Thr 
                645                 650                 655     


Phe Gln Ile Ser Gln Thr Asp Leu Phe Gly Asn Pro Met Met Tyr Asp 
            660                 665                 670         


Leu Lys Glu Asn Gly Asp Lys Ile Pro Ile Thr Asn Glu Asn Arg Lys 
        675                 680                 685             


Glu Phe Val Asn Leu Tyr Ser Asp Tyr Ile Leu Asn Lys Ser Val Glu 
    690                 695                 700                 


Lys Gln Phe Lys Ala Phe Arg Arg Gly Phe His Met Val Thr Asn Glu 
705                 710                 715                 720 


Ser Pro Leu Lys Tyr Leu Phe Arg Pro Glu Glu Ile Glu Leu Leu Ile 
                725                 730                 735     


Cys Gly Ser Arg Asn Leu Asp Phe Gln Ala Leu Glu Glu Thr Thr Glu 
            740                 745                 750         


Tyr Asp Gly Gly Tyr Thr Arg Asp Ser Val Leu Ile Arg Glu Phe Trp 
        755                 760                 765             


Glu Ile Val His Ser Phe Thr Asp Glu Gln Lys Arg Leu Phe Leu Gln 
    770                 775                 780                 


Phe Thr Thr Gly Thr Asp Arg Ala Pro Val Gly Gly Leu Gly Lys Leu 
785                 790                 795                 800 


Lys Met Ile Ile Ala Lys Asn Gly Pro Asp Thr Glu Arg Leu Pro Thr 
                805                 810                 815     


Ser His Thr Cys Phe Asn Val Leu Leu Leu Pro Glu Tyr Ser Ser Lys 
            820                 825                 830         


Glu Lys Leu Lys Glu Arg Leu Leu Lys Ala Ile Thr Tyr Ala Lys Gly 
        835                 840                 845             


Phe Gly Met Leu 
    850         


<210>  3
<211>  3700
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector genome hSyn.hUbe3a-1.GSco.SV40


<220>
<221>  repeat_region
<222>  (1)..(130)
<223>  ITR

<220>
<221>  promoter
<222>  (213)..(678)
<223>  hSynapsin promoter

<220>
<221>  misc_feature
<222>  (690)..(695)
<223>  Kozak

<220>
<221>  CDS
<222>  (696)..(3257)
<223>  hUBE3a-1

<220>
<221>  misc_feature
<222>  (779)..(779)
<223>  mut

<220>
<221>  misc_feature
<222>  (824)..(824)
<223>  mut

<220>
<221>  misc_feature
<222>  (1028)..(1028)
<223>  mut

<220>
<221>  misc_feature
<222>  (1181)..(1181)
<223>  mut

<220>
<221>  misc_feature
<222>  (1193)..(1193)
<223>  mut

<220>
<221>  misc_feature
<222>  (1217)..(1217)
<223>  mut

<220>
<221>  misc_feature
<222>  (1283)..(1283)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (1715)..(1715)
<223>  mut

<220>
<221>  misc_feature
<222>  (1721)..(1721)
<223>  mut

<220>
<221>  misc_feature
<222>  (1766)..(1766)
<223>  mut

<220>
<221>  misc_feature
<222>  (1805)..(1805)
<223>  mut

<220>
<221>  misc_feature
<222>  (1814)..(1814)
<223>  mut

<220>
<221>  misc_feature
<222>  (1991)..(1991)
<223>  mut

<220>
<221>  misc_feature
<222>  (2063)..(2063)
<223>  mut

<220>
<221>  misc_feature
<222>  (2111)..(2111)
<223>  mut

<220>
<221>  misc_feature
<222>  (2294)..(2294)
<223>  mut

<220>
<221>  misc_feature
<222>  (2439)..(2440)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (2723)..(2723)
<223>  mut

<220>
<221>  misc_feature
<222>  (2957)..(2957)
<223>  mut

<220>
<221>  misc_feature
<222>  (3023)..(3023)
<223>  mut

<220>
<221>  misc_feature
<222>  (3056)..(3056)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (3152)..(3152)
<223>  mut

<220>
<221>  misc_feature
<222>  (3161)..(3161)
<223>  mut

<220>
<221>  misc_feature
<222>  (3230)..(3230)
<223>  mut

<220>
<221>  polyA_signal
<222>  (3275)..(3700)
<223>  SV40 polyA

<220>
<221>  repeat_region
<222>  (3571)..(3700)
<223>  ITR

<400>  3
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct tgtagttaat gattaacccg ccatgctact tatctacgta gccatgctct      180

aggaagatcc tctagaacta tagctagcat gcctgcagag ggccctgcgt atgagtgcaa      240

gtgggtttta ggaccaggat gaggcggggt gggggtgcct acctgacgac cgaccccgac      300

ccactggaca agcacccaac ccccattccc caaattgcgc atcccctatc agagaggggg      360

aggggaaaca ggatgcggcg aggcgcgtgc gcactgccag cttcagcacc gcggacagtg      420

ccttcgcccc cgcctggcgg cgcgcgccac cgccgcctca gcactgaagg cgcgctgacg      480

tcactcgccg gtcccccgca aactcccctt cccggccacc ttggtcgcgt ccgcgccgcc      540

gccggcccag ccggaccgca ccacgcgagg cgcgagatag gggggcacgg gcgcgaccat      600

ctgcgctgcg gcgccggcga ctcagcgctg cctcagtctg cggtgggcag cggaggagtc      660

gtgtcgtgcc tgagagcgca gtcgaattcg ccacc atg aag agg gca gca gca         713
                                       Met Lys Arg Ala Ala Ala            
                                       1               5                  

aag cac ctg atc gag aga tac tat cac cag ctg acc gag gga tgc gga        761
Lys His Leu Ile Glu Arg Tyr Tyr His Gln Leu Thr Glu Gly Cys Gly           
            10                  15                  20                    

aac gag gca tgt aca aac gag ttc tgc gcc tcc tgt ccc acc ttt ctg        809
Asn Glu Ala Cys Thr Asn Glu Phe Cys Ala Ser Cys Pro Thr Phe Leu           
        25                  30                  35                        

agg atg gat aac aac gcc gcc gcc atc aag gcc ctg gag ctg tac aag        857
Arg Met Asp Asn Asn Ala Ala Ala Ile Lys Ala Leu Glu Leu Tyr Lys           
    40                  45                  50                            

atc aac gcc aag ctg tgc gac ccc cac cct agc aag aag ggc gcc agc        905
Ile Asn Ala Lys Leu Cys Asp Pro His Pro Ser Lys Lys Gly Ala Ser           
55                  60                  65                  70            

tcc gcc tat ctg gag aac tcc aag ggc gcc cct aac aat agc tgt tcc        953
Ser Ala Tyr Leu Glu Asn Ser Lys Gly Ala Pro Asn Asn Ser Cys Ser           
                75                  80                  85                

gag atc aag atg aat aag aag ggc gcc cgg atc gat ttc aag gac gtg       1001
Glu Ile Lys Met Asn Lys Lys Gly Ala Arg Ile Asp Phe Lys Asp Val           
            90                  95                  100                   

acc tac ctg aca gag gag aag gtg tac gag atc ctg gag ctg tgc cgg       1049
Thr Tyr Leu Thr Glu Glu Lys Val Tyr Glu Ile Leu Glu Leu Cys Arg           
        105                 110                 115                       

gag aga gag gat tac agc cca ctg atc aga gtg atc ggc aga gtg ttc       1097
Glu Arg Glu Asp Tyr Ser Pro Leu Ile Arg Val Ile Gly Arg Val Phe           
    120                 125                 130                           

tct agc gcc gag gcc ctg gtg cag tcc ttt aga aag gtg aag cag cac       1145
Ser Ser Ala Glu Ala Leu Val Gln Ser Phe Arg Lys Val Lys Gln His           
135                 140                 145                 150           

aca aag gag gag ctg aag tct ctg cag gcc aag gac gag gac aag gac       1193
Thr Lys Glu Glu Leu Lys Ser Leu Gln Ala Lys Asp Glu Asp Lys Asp           
                155                 160                 165               

gag gac gag aag gag aag gca gcc tgt tct gcc gca gca atg gag gag       1241
Glu Asp Glu Lys Glu Lys Ala Ala Cys Ser Ala Ala Ala Met Glu Glu           
            170                 175                 180                   

gac agc gag gca tcc tct agc cgg atc ggc gat tcc tct caa ggc gac       1289
Asp Ser Glu Ala Ser Ser Ser Arg Ile Gly Asp Ser Ser Gln Gly Asp           
        185                 190                 195                       

aac aat ctg cag aag ctg ggc ccc gac gac gtg agc gtg gat atc gac       1337
Asn Asn Leu Gln Lys Leu Gly Pro Asp Asp Val Ser Val Asp Ile Asp           
    200                 205                 210                           

gcc atc cgg aga gtg tac aca aga ctg ctg agc aac gag aag atc gag       1385
Ala Ile Arg Arg Val Tyr Thr Arg Leu Leu Ser Asn Glu Lys Ile Glu           
215                 220                 225                 230           

acc gcc ttc ctg aac gcc ctg gtg tat ctg agc cct aat gtg gag tgc       1433
Thr Ala Phe Leu Asn Ala Leu Val Tyr Leu Ser Pro Asn Val Glu Cys           
                235                 240                 245               

gat ctg acc tac cac aac gtg tac tcc cgg gac cca aac tac ctg aat       1481
Asp Leu Thr Tyr His Asn Val Tyr Ser Arg Asp Pro Asn Tyr Leu Asn           
            250                 255                 260                   

ctg ttc atc atc gtg atg gag aac aga aat ctg cac tcc ccc gag tat       1529
Leu Phe Ile Ile Val Met Glu Asn Arg Asn Leu His Ser Pro Glu Tyr           
        265                 270                 275                       

ctg gag atg gcc ctg cct ctg ttt tgt aag gcc atg tcc aag ctg cct       1577
Leu Glu Met Ala Leu Pro Leu Phe Cys Lys Ala Met Ser Lys Leu Pro           
    280                 285                 290                           

ctg gca gca cag ggc aag ctg atc agg ctg tgg tct aag tac aac gcc       1625
Leu Ala Ala Gln Gly Lys Leu Ile Arg Leu Trp Ser Lys Tyr Asn Ala           
295                 300                 305                 310           

gat cag atc agg cgc atg atg gag acc ttc cag cag ctg atc aca tac       1673
Asp Gln Ile Arg Arg Met Met Glu Thr Phe Gln Gln Leu Ile Thr Tyr           
                315                 320                 325               

aaa gtg atc tct aac gag ttt aat agc cgc aac ctg gtg aac gac gac       1721
Lys Val Ile Ser Asn Glu Phe Asn Ser Arg Asn Leu Val Asn Asp Asp           
            330                 335                 340                   

gac gcc atc gtg gcc gcc tct aag tgc ctg aag atg gtg tac tac gcc       1769
Asp Ala Ile Val Ala Ala Ser Lys Cys Leu Lys Met Val Tyr Tyr Ala           
        345                 350                 355                       

aac gtg gtg ggc ggc gag gtg gac aca aac cac aac gag gag gac gac       1817
Asn Val Val Gly Gly Glu Val Asp Thr Asn His Asn Glu Glu Asp Asp           
    360                 365                 370                           

gag gag cca atc ccc gag agc tcc gag ctg acc ctg cag gag ctg ctg       1865
Glu Glu Pro Ile Pro Glu Ser Ser Glu Leu Thr Leu Gln Glu Leu Leu           
375                 380                 385                 390           

gga gag gag cgg aga aat aag aag gga cca agg gtg gat cct ctg gag       1913
Gly Glu Glu Arg Arg Asn Lys Lys Gly Pro Arg Val Asp Pro Leu Glu           
                395                 400                 405               

acc gag ctg ggc gtg aag aca ctg gac tgc aga aag cct ctg atc cca       1961
Thr Glu Leu Gly Val Lys Thr Leu Asp Cys Arg Lys Pro Leu Ile Pro           
            410                 415                 420                   

ttc gag gag ttt atc aac gag ccc ctg aac gag gtg ctg gag atg gat       2009
Phe Glu Glu Phe Ile Asn Glu Pro Leu Asn Glu Val Leu Glu Met Asp           
        425                 430                 435                       

aag gac tac acc ttc ttt aag gtg gag aca gag aac aag ttc agc ttt       2057
Lys Asp Tyr Thr Phe Phe Lys Val Glu Thr Glu Asn Lys Phe Ser Phe           
    440                 445                 450                           

atg acc tgt cct ttc atc ctg aac gcc gtg acc aag aat ctg ggc ctg       2105
Met Thr Cys Pro Phe Ile Leu Asn Ala Val Thr Lys Asn Leu Gly Leu           
455                 460                 465                 470           

tac tac gat aac agg atc cgc atg tac tcc gag agg cgc atc acc gtg       2153
Tyr Tyr Asp Asn Arg Ile Arg Met Tyr Ser Glu Arg Arg Ile Thr Val           
                475                 480                 485               

ctg tat tct ctg gtg cag ggc cag cag ctg aat cct tac ctg agg ctg       2201
Leu Tyr Ser Leu Val Gln Gly Gln Gln Leu Asn Pro Tyr Leu Arg Leu           
            490                 495                 500                   

aag gtg cgg aga gac cac atc atc gat gac gcc ctg gtg cgc ctg gag       2249
Lys Val Arg Arg Asp His Ile Ile Asp Asp Ala Leu Val Arg Leu Glu           
        505                 510                 515                       

atg atc gcc atg gag aat cca gcc gat ctg aag aag cag ctg tac gtg       2297
Met Ile Ala Met Glu Asn Pro Ala Asp Leu Lys Lys Gln Leu Tyr Val           
    520                 525                 530                           

gag ttt gag gga gag cag gga gtg gac gag gga ggc gtg tct aag gag       2345
Glu Phe Glu Gly Glu Gln Gly Val Asp Glu Gly Gly Val Ser Lys Glu           
535                 540                 545                 550           

ttc ttt cag ctg gtg gtg gag gag atc ttc aac ccc gat atc ggc atg       2393
Phe Phe Gln Leu Val Val Glu Glu Ile Phe Asn Pro Asp Ile Gly Met           
                555                 560                 565               

ttt acc tac gac gag agc aca aag ctg ttc tgg ttt aat cct tct tcc       2441
Phe Thr Tyr Asp Glu Ser Thr Lys Leu Phe Trp Phe Asn Pro Ser Ser           
            570                 575                 580                   

ttc gag acc gag ggc cag ttt aca ctg atc ggc atc gtg ctg ggc ctg       2489
Phe Glu Thr Glu Gly Gln Phe Thr Leu Ile Gly Ile Val Leu Gly Leu           
        585                 590                 595                       

gcc atc tac aac aat tgt atc ctg gac gtg cac ttc cca atg gtg gtg       2537
Ala Ile Tyr Asn Asn Cys Ile Leu Asp Val His Phe Pro Met Val Val           
    600                 605                 610                           

tat agg aag ctg atg ggc aag aag ggc acc ttt cgc gat ctg ggc gac       2585
Tyr Arg Lys Leu Met Gly Lys Lys Gly Thr Phe Arg Asp Leu Gly Asp           
615                 620                 625                 630           

tcc cac ccc gtg ctg tac cag tct ctg aag gat ctg ctg gag tat gag       2633
Ser His Pro Val Leu Tyr Gln Ser Leu Lys Asp Leu Leu Glu Tyr Glu           
                635                 640                 645               

ggc aac gtg gag gat gac atg atg atc acc ttc cag atc tcc cag aca       2681
Gly Asn Val Glu Asp Asp Met Met Ile Thr Phe Gln Ile Ser Gln Thr           
            650                 655                 660                   

gac ctg ttt ggc aac cca atg atg tac gat ctg aag gag aac ggc gac       2729
Asp Leu Phe Gly Asn Pro Met Met Tyr Asp Leu Lys Glu Asn Gly Asp           
        665                 670                 675                       

aag atc ccc atc aca aac gag aat aga aag gag ttc gtg aac ctg tac       2777
Lys Ile Pro Ile Thr Asn Glu Asn Arg Lys Glu Phe Val Asn Leu Tyr           
    680                 685                 690                           

agc gat tat atc ctg aat aag tcc gtg gag aag cag ttc aag gcc ttt       2825
Ser Asp Tyr Ile Leu Asn Lys Ser Val Glu Lys Gln Phe Lys Ala Phe           
695                 700                 705                 710           

agg cgc ggc ttc cac atg gtg acc aac gag agc cct ctg aag tat ctg       2873
Arg Arg Gly Phe His Met Val Thr Asn Glu Ser Pro Leu Lys Tyr Leu           
                715                 720                 725               

ttt agg cca gag gag atc gag ctg ctg atc tgc ggc tcc cgc aat ctg       2921
Phe Arg Pro Glu Glu Ile Glu Leu Leu Ile Cys Gly Ser Arg Asn Leu           
            730                 735                 740                   

gac ttt cag gcc ctg gag gag acc aca gag tac gac ggc ggc tat acc       2969
Asp Phe Gln Ala Leu Glu Glu Thr Thr Glu Tyr Asp Gly Gly Tyr Thr           
        745                 750                 755                       

agg gac tct gtg ctg atc cgc gag ttc tgg gag atc gtg cac agc ttt       3017
Arg Asp Ser Val Leu Ile Arg Glu Phe Trp Glu Ile Val His Ser Phe           
    760                 765                 770                           

aca gac gag cag aag cgg ctg ttc ctg cag ttt acc acc ggc acc gac       3065
Thr Asp Glu Gln Lys Arg Leu Phe Leu Gln Phe Thr Thr Gly Thr Asp           
775                 780                 785                 790           

aga gca cca gtg gga gga ctg ggc aag ctg aag atg atc atc gcc aag       3113
Arg Ala Pro Val Gly Gly Leu Gly Lys Leu Lys Met Ile Ile Ala Lys           
                795                 800                 805               

aac ggc cca gac aca gag agg ctg ccc acc agc cac acc tgt ttc aac       3161
Asn Gly Pro Asp Thr Glu Arg Leu Pro Thr Ser His Thr Cys Phe Asn           
            810                 815                 820                   

gtg ctg ctg ctg ccc gag tac tcc tct aag gag aag ctg aag gag cgc       3209
Val Leu Leu Leu Pro Glu Tyr Ser Ser Lys Glu Lys Leu Lys Glu Arg           
        825                 830                 835                       

ctg ctg aag gcc atc acc tac gcc aag ggc ttt ggc atg ctg tga tga       3257
Leu Leu Lys Ala Ile Thr Tyr Ala Lys Gly Phe Gly Met Leu                   
    840                 845                 850                           

ggtaccggcg gccgcttcga gcagacatga taagatacat tgatgagttt ggacaaacca     3317

caactagaat gcagtgaaaa aaatgcttta tttgtgaaat ttgtgatgct attgctttat     3377

ttgtaaccat tataagctgc aataaacaag ttaacaacaa caattgcatt cattttatgt     3437

ttcaggttca gggggagatg tgggaggttt tttaaagcaa gtaaaacctc tacaaatgtg     3497

gtaaaatcga taaggatctt cctagagcat ggctacgtag ataagtagca tggcgggtta     3557

atcattaact acaaggaacc cctagtgatg gagttggcca ctccctctct gcgcgctcgc     3617

tcgctcactg aggccgggcg accaaaggtc gcccgacgcc cgggctttgc ccgggcggcc     3677

tcagtgagcg agcgagcgcg cag                                             3700


<210>  4
<211>  852
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Synthetic Construct

<400>  4

Met Lys Arg Ala Ala Ala Lys His Leu Ile Glu Arg Tyr Tyr His Gln 
1               5                   10                  15      


Leu Thr Glu Gly Cys Gly Asn Glu Ala Cys Thr Asn Glu Phe Cys Ala 
            20                  25                  30          


Ser Cys Pro Thr Phe Leu Arg Met Asp Asn Asn Ala Ala Ala Ile Lys 
        35                  40                  45              


Ala Leu Glu Leu Tyr Lys Ile Asn Ala Lys Leu Cys Asp Pro His Pro 
    50                  55                  60                  


Ser Lys Lys Gly Ala Ser Ser Ala Tyr Leu Glu Asn Ser Lys Gly Ala 
65                  70                  75                  80  


Pro Asn Asn Ser Cys Ser Glu Ile Lys Met Asn Lys Lys Gly Ala Arg 
                85                  90                  95      


Ile Asp Phe Lys Asp Val Thr Tyr Leu Thr Glu Glu Lys Val Tyr Glu 
            100                 105                 110         


Ile Leu Glu Leu Cys Arg Glu Arg Glu Asp Tyr Ser Pro Leu Ile Arg 
        115                 120                 125             


Val Ile Gly Arg Val Phe Ser Ser Ala Glu Ala Leu Val Gln Ser Phe 
    130                 135                 140                 


Arg Lys Val Lys Gln His Thr Lys Glu Glu Leu Lys Ser Leu Gln Ala 
145                 150                 155                 160 


Lys Asp Glu Asp Lys Asp Glu Asp Glu Lys Glu Lys Ala Ala Cys Ser 
                165                 170                 175     


Ala Ala Ala Met Glu Glu Asp Ser Glu Ala Ser Ser Ser Arg Ile Gly 
            180                 185                 190         


Asp Ser Ser Gln Gly Asp Asn Asn Leu Gln Lys Leu Gly Pro Asp Asp 
        195                 200                 205             


Val Ser Val Asp Ile Asp Ala Ile Arg Arg Val Tyr Thr Arg Leu Leu 
    210                 215                 220                 


Ser Asn Glu Lys Ile Glu Thr Ala Phe Leu Asn Ala Leu Val Tyr Leu 
225                 230                 235                 240 


Ser Pro Asn Val Glu Cys Asp Leu Thr Tyr His Asn Val Tyr Ser Arg 
                245                 250                 255     


Asp Pro Asn Tyr Leu Asn Leu Phe Ile Ile Val Met Glu Asn Arg Asn 
            260                 265                 270         


Leu His Ser Pro Glu Tyr Leu Glu Met Ala Leu Pro Leu Phe Cys Lys 
        275                 280                 285             


Ala Met Ser Lys Leu Pro Leu Ala Ala Gln Gly Lys Leu Ile Arg Leu 
    290                 295                 300                 


Trp Ser Lys Tyr Asn Ala Asp Gln Ile Arg Arg Met Met Glu Thr Phe 
305                 310                 315                 320 


Gln Gln Leu Ile Thr Tyr Lys Val Ile Ser Asn Glu Phe Asn Ser Arg 
                325                 330                 335     


Asn Leu Val Asn Asp Asp Asp Ala Ile Val Ala Ala Ser Lys Cys Leu 
            340                 345                 350         


Lys Met Val Tyr Tyr Ala Asn Val Val Gly Gly Glu Val Asp Thr Asn 
        355                 360                 365             


His Asn Glu Glu Asp Asp Glu Glu Pro Ile Pro Glu Ser Ser Glu Leu 
    370                 375                 380                 


Thr Leu Gln Glu Leu Leu Gly Glu Glu Arg Arg Asn Lys Lys Gly Pro 
385                 390                 395                 400 


Arg Val Asp Pro Leu Glu Thr Glu Leu Gly Val Lys Thr Leu Asp Cys 
                405                 410                 415     


Arg Lys Pro Leu Ile Pro Phe Glu Glu Phe Ile Asn Glu Pro Leu Asn 
            420                 425                 430         


Glu Val Leu Glu Met Asp Lys Asp Tyr Thr Phe Phe Lys Val Glu Thr 
        435                 440                 445             


Glu Asn Lys Phe Ser Phe Met Thr Cys Pro Phe Ile Leu Asn Ala Val 
    450                 455                 460                 


Thr Lys Asn Leu Gly Leu Tyr Tyr Asp Asn Arg Ile Arg Met Tyr Ser 
465                 470                 475                 480 


Glu Arg Arg Ile Thr Val Leu Tyr Ser Leu Val Gln Gly Gln Gln Leu 
                485                 490                 495     


Asn Pro Tyr Leu Arg Leu Lys Val Arg Arg Asp His Ile Ile Asp Asp 
            500                 505                 510         


Ala Leu Val Arg Leu Glu Met Ile Ala Met Glu Asn Pro Ala Asp Leu 
        515                 520                 525             


Lys Lys Gln Leu Tyr Val Glu Phe Glu Gly Glu Gln Gly Val Asp Glu 
    530                 535                 540                 


Gly Gly Val Ser Lys Glu Phe Phe Gln Leu Val Val Glu Glu Ile Phe 
545                 550                 555                 560 


Asn Pro Asp Ile Gly Met Phe Thr Tyr Asp Glu Ser Thr Lys Leu Phe 
                565                 570                 575     


Trp Phe Asn Pro Ser Ser Phe Glu Thr Glu Gly Gln Phe Thr Leu Ile 
            580                 585                 590         


Gly Ile Val Leu Gly Leu Ala Ile Tyr Asn Asn Cys Ile Leu Asp Val 
        595                 600                 605             


His Phe Pro Met Val Val Tyr Arg Lys Leu Met Gly Lys Lys Gly Thr 
    610                 615                 620                 


Phe Arg Asp Leu Gly Asp Ser His Pro Val Leu Tyr Gln Ser Leu Lys 
625                 630                 635                 640 


Asp Leu Leu Glu Tyr Glu Gly Asn Val Glu Asp Asp Met Met Ile Thr 
                645                 650                 655     


Phe Gln Ile Ser Gln Thr Asp Leu Phe Gly Asn Pro Met Met Tyr Asp 
            660                 665                 670         


Leu Lys Glu Asn Gly Asp Lys Ile Pro Ile Thr Asn Glu Asn Arg Lys 
        675                 680                 685             


Glu Phe Val Asn Leu Tyr Ser Asp Tyr Ile Leu Asn Lys Ser Val Glu 
    690                 695                 700                 


Lys Gln Phe Lys Ala Phe Arg Arg Gly Phe His Met Val Thr Asn Glu 
705                 710                 715                 720 


Ser Pro Leu Lys Tyr Leu Phe Arg Pro Glu Glu Ile Glu Leu Leu Ile 
                725                 730                 735     


Cys Gly Ser Arg Asn Leu Asp Phe Gln Ala Leu Glu Glu Thr Thr Glu 
            740                 745                 750         


Tyr Asp Gly Gly Tyr Thr Arg Asp Ser Val Leu Ile Arg Glu Phe Trp 
        755                 760                 765             


Glu Ile Val His Ser Phe Thr Asp Glu Gln Lys Arg Leu Phe Leu Gln 
    770                 775                 780                 


Phe Thr Thr Gly Thr Asp Arg Ala Pro Val Gly Gly Leu Gly Lys Leu 
785                 790                 795                 800 


Lys Met Ile Ile Ala Lys Asn Gly Pro Asp Thr Glu Arg Leu Pro Thr 
                805                 810                 815     


Ser His Thr Cys Phe Asn Val Leu Leu Leu Pro Glu Tyr Ser Ser Lys 
            820                 825                 830         


Glu Lys Leu Lys Glu Arg Leu Leu Lys Ala Ile Thr Tyr Ala Lys Gly 
        835                 840                 845             


Phe Gly Met Leu 
    850         


<210>  5
<211>  3873
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector genome hSyn.hUbe3a-2.GSco.4XmiRNA183.SV40


<220>
<221>  repeat_region
<222>  (1)..(130)
<223>  ITR

<220>
<221>  promoter
<222>  (213)..(678)
<223>  hSynapsin promoter

<220>
<221>  misc_feature
<222>  (690)..(695)
<223>  Kozak

<220>
<221>  CDS
<222>  (696)..(3326)
<223>  hUBE3a-2

<220>
<221>  misc_feature
<222>  (848)..(848)
<223>  mut

<220>
<221>  misc_feature
<222>  (893)..(893)
<223>  mut

<220>
<221>  misc_feature
<222>  (1097)..(1097)
<223>  mut

<220>
<221>  misc_feature
<222>  (1250)..(1250)
<223>  mut

<220>
<221>  misc_feature
<222>  (1262)..(1262)
<223>  mut

<220>
<221>  misc_feature
<222>  (1286)..(1286)
<223>  mut

<220>
<221>  misc_feature
<222>  (1352)..(1352)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (1784)..(1784)
<223>  mut

<220>
<221>  misc_feature
<222>  (1790)..(1790)
<223>  mut

<220>
<221>  misc_feature
<222>  (1835)..(1835)
<223>  mut

<220>
<221>  misc_feature
<222>  (1874)..(1874)
<223>  mut

<220>
<221>  misc_feature
<222>  (1883)..(1883)
<223>  mut

<220>
<221>  misc_feature
<222>  (2060)..(2060)
<223>  mut

<220>
<221>  misc_feature
<222>  (2132)..(2132)
<223>  mut

<220>
<221>  misc_feature
<222>  (2180)..(2180)
<223>  mut

<220>
<221>  misc_feature
<222>  (2363)..(2363)
<223>  mut

<220>
<221>  misc_feature
<222>  (2508)..(2508)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (2792)..(2792)
<223>  mut

<220>
<221>  misc_feature
<222>  (3026)..(3026)
<223>  mut

<220>
<221>  misc_feature
<222>  (3092)..(3092)
<223>  mut

<220>
<221>  misc_feature
<222>  (3125)..(3125)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (3221)..(3221)
<223>  mut

<220>
<221>  misc_feature
<222>  (3230)..(3230)
<223>  mut

<220>
<221>  misc_feature
<222>  (3299)..(3299)
<223>  mut

<220>
<221>  misc_feature
<222>  (3333)..(3354)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3359)..(3380)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3387)..(3408)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3415)..(3436)
<223>  miRNA183

<220>
<221>  polyA_signal
<222>  (3448)..(3679)
<223>  SV40 polyA

<220>
<221>  repeat_region
<222>  (3744)..(3873)
<223>  ITR

<400>  5
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct tgtagttaat gattaacccg ccatgctact tatctacgta gccatgctct      180

aggaagatcc tctagaacta tagctagcat gcctgcagag ggccctgcgt atgagtgcaa      240

gtgggtttta ggaccaggat gaggcggggt gggggtgcct acctgacgac cgaccccgac      300

ccactggaca agcacccaac ccccattccc caaattgcgc atcccctatc agagaggggg      360

aggggaaaca ggatgcggcg aggcgcgtgc gcactgccag cttcagcacc gcggacagtg      420

ccttcgcccc cgcctggcgg cgcgcgccac cgccgcctca gcactgaagg cgcgctgacg      480

tcactcgccg gtcccccgca aactcccctt cccggccacc ttggtcgcgt ccgcgccgcc      540

gccggcccag ccggaccgca ccacgcgagg cgcgagatag gggggcacgg gcgcgaccat      600

ctgcgctgcg gcgccggcga ctcagcgctg cctcagtctg cggtgggcag cggaggagtc      660

gtgtcgtgcc tgagagcgca gtcgaattcg ccacc atg gag aag ctg cac cag         713
                                       Met Glu Lys Leu His Gln            
                                       1               5                  

tgc tac tgg aag tct ggc gag cct cag agc gac gat atc gag gca agc        761
Cys Tyr Trp Lys Ser Gly Glu Pro Gln Ser Asp Asp Ile Glu Ala Ser           
            10                  15                  20                    

agg atg aag aga gca gca gcc aag cac ctg atc gag cgg tac tat cac        809
Arg Met Lys Arg Ala Ala Ala Lys His Leu Ile Glu Arg Tyr Tyr His           
        25                  30                  35                        

cag ctg acc gag ggc tgc gga aac gag gcc tgt aca aac gag ttc tgc        857
Gln Leu Thr Glu Gly Cys Gly Asn Glu Ala Cys Thr Asn Glu Phe Cys           
    40                  45                  50                            

gcc tcc tgt cca acc ttt ctg aga atg gat aac aac gcc gcc gcc atc        905
Ala Ser Cys Pro Thr Phe Leu Arg Met Asp Asn Asn Ala Ala Ala Ile           
55                  60                  65                  70            

aag gcc ctg gag ctg tac aag atc aac gcc aag ctg tgc gac ccc cac        953
Lys Ala Leu Glu Leu Tyr Lys Ile Asn Ala Lys Leu Cys Asp Pro His           
                75                  80                  85                

cct tct aag aag ggc gcc agc tcc gcc tat ctg gag aac agc aag ggc       1001
Pro Ser Lys Lys Gly Ala Ser Ser Ala Tyr Leu Glu Asn Ser Lys Gly           
            90                  95                  100                   

gcc ccc aac aat agc tgt tcc gag atc aag atg aat aag aag ggc gcc       1049
Ala Pro Asn Asn Ser Cys Ser Glu Ile Lys Met Asn Lys Lys Gly Ala           
        105                 110                 115                       

agg atc gat ttc aag gac gtg acc tac ctg aca gag gag aag gtg tac       1097
Arg Ile Asp Phe Lys Asp Val Thr Tyr Leu Thr Glu Glu Lys Val Tyr           
    120                 125                 130                           

gag atc ctg gag ctg tgc cgg gag aga gag gat tac tcc cct ctg atc       1145
Glu Ile Leu Glu Leu Cys Arg Glu Arg Glu Asp Tyr Ser Pro Leu Ile           
135                 140                 145                 150           

aga gtg atc gga cgc gtg ttc tct agc gcc gag gcc ctg gtg cag agc       1193
Arg Val Ile Gly Arg Val Phe Ser Ser Ala Glu Ala Leu Val Gln Ser           
                155                 160                 165               

ttt cgc aag gtg aag cag cac aca aag gag gag ctg aag tcc ctg cag       1241
Phe Arg Lys Val Lys Gln His Thr Lys Glu Glu Leu Lys Ser Leu Gln           
            170                 175                 180                   

gcc aag gac gag gac aag gac gag gac gag aag gag aag gca gct tgt       1289
Ala Lys Asp Glu Asp Lys Asp Glu Asp Glu Lys Glu Lys Ala Ala Cys           
        185                 190                 195                       

tcc gcc gca gca atg gag gag gac tct gag gcc tcc tct agc agg atc       1337
Ser Ala Ala Ala Met Glu Glu Asp Ser Glu Ala Ser Ser Ser Arg Ile           
    200                 205                 210                           

ggc gat tcc tct caa ggc gac aac aat ctg cag aag ctg ggc ccc gac       1385
Gly Asp Ser Ser Gln Gly Asp Asn Asn Leu Gln Lys Leu Gly Pro Asp           
215                 220                 225                 230           

gac gtg agc gtg gat atc gac gcc atc cgg aga gtg tac aca cgc ctg       1433
Asp Val Ser Val Asp Ile Asp Ala Ile Arg Arg Val Tyr Thr Arg Leu           
                235                 240                 245               

ctg tct aac gag aag atc gag acc gcc ttc ctg aac gcc ctg gtg tat       1481
Leu Ser Asn Glu Lys Ile Glu Thr Ala Phe Leu Asn Ala Leu Val Tyr           
            250                 255                 260                   

ctg tct cct aat gtg gag tgc gat ctg acc tac cac aac gtg tac agc       1529
Leu Ser Pro Asn Val Glu Cys Asp Leu Thr Tyr His Asn Val Tyr Ser           
        265                 270                 275                       

cgg gac cca aac tac ctg aat ctg ttc atc atc gtg atg gag aac aga       1577
Arg Asp Pro Asn Tyr Leu Asn Leu Phe Ile Ile Val Met Glu Asn Arg           
    280                 285                 290                           

aat ctg cac tct ccc gag tat ctg gag atg gcc ctg cct ctg ttt tgt       1625
Asn Leu His Ser Pro Glu Tyr Leu Glu Met Ala Leu Pro Leu Phe Cys           
295                 300                 305                 310           

aag gcc atg agc aag ctg cca ctg gca gca cag ggc aag ctg atc agg       1673
Lys Ala Met Ser Lys Leu Pro Leu Ala Ala Gln Gly Lys Leu Ile Arg           
                315                 320                 325               

ctg tgg tcc aag tac aac gcc gat cag atc agg cgc atg atg gag acc       1721
Leu Trp Ser Lys Tyr Asn Ala Asp Gln Ile Arg Arg Met Met Glu Thr           
            330                 335                 340                   

ttc cag cag ctg atc aca tat aaa gtg atc tcc aac gag ttt aat tct       1769
Phe Gln Gln Leu Ile Thr Tyr Lys Val Ile Ser Asn Glu Phe Asn Ser           
        345                 350                 355                       

aga aac ctg gtg aac gac gac gac gcc atc gtg gcc gcc agc aag tgc       1817
Arg Asn Leu Val Asn Asp Asp Asp Ala Ile Val Ala Ala Ser Lys Cys           
    360                 365                 370                           

ctg aag atg gtg tac tac gcc aac gtg gtg ggc ggc gag gtg gac aca       1865
Leu Lys Met Val Tyr Tyr Ala Asn Val Val Gly Gly Glu Val Asp Thr           
375                 380                 385                 390           

aac cac aac gag gag gac gac gag gag cca atc ccc gag agc tcc gag       1913
Asn His Asn Glu Glu Asp Asp Glu Glu Pro Ile Pro Glu Ser Ser Glu           
                395                 400                 405               

ctg acc ctg cag gag ctg ctg gga gag gag cgg aga aat aag aag gga       1961
Leu Thr Leu Gln Glu Leu Leu Gly Glu Glu Arg Arg Asn Lys Lys Gly           
            410                 415                 420                   

cca agg gtg gat cct ctg gag acc gag ctg ggc gtg aag aca ctg gac       2009
Pro Arg Val Asp Pro Leu Glu Thr Glu Leu Gly Val Lys Thr Leu Asp           
        425                 430                 435                       

tgc aga aag cct ctg atc cca ttc gag gag ttt atc aac gag ccc ctg       2057
Cys Arg Lys Pro Leu Ile Pro Phe Glu Glu Phe Ile Asn Glu Pro Leu           
    440                 445                 450                           

aac gag gtg ctg gag atg gat aag gac tac acc ttc ttt aag gtg gag       2105
Asn Glu Val Leu Glu Met Asp Lys Asp Tyr Thr Phe Phe Lys Val Glu           
455                 460                 465                 470           

aca gag aac aag ttc agc ttt atg acc tgt cct ttc atc ctg aac gcc       2153
Thr Glu Asn Lys Phe Ser Phe Met Thr Cys Pro Phe Ile Leu Asn Ala           
                475                 480                 485               

gtg acc aag aat ctg ggc ctg tac tac gat aac agg atc cgc atg tac       2201
Val Thr Lys Asn Leu Gly Leu Tyr Tyr Asp Asn Arg Ile Arg Met Tyr           
            490                 495                 500                   

agc gag agg cgc atc acc gtg ctg tat tcc ctg gtg cag ggc cag cag       2249
Ser Glu Arg Arg Ile Thr Val Leu Tyr Ser Leu Val Gln Gly Gln Gln           
        505                 510                 515                       

ctg aat cct tac ctg agg ctg aag gtg cgg aga gac cac atc atc gat       2297
Leu Asn Pro Tyr Leu Arg Leu Lys Val Arg Arg Asp His Ile Ile Asp           
    520                 525                 530                           

gac gcc ctg gtg cgc ctg gag atg atc gcc atg gag aat cca gcc gat       2345
Asp Ala Leu Val Arg Leu Glu Met Ile Ala Met Glu Asn Pro Ala Asp           
535                 540                 545                 550           

ctg aag aag cag ctg tac gtg gag ttt gag gga gag cag gga gtg gac       2393
Leu Lys Lys Gln Leu Tyr Val Glu Phe Glu Gly Glu Gln Gly Val Asp           
                555                 560                 565               

gag gga ggc gtg tcc aag gag ttc ttt cag ctg gtg gtg gag gag atc       2441
Glu Gly Gly Val Ser Lys Glu Phe Phe Gln Leu Val Val Glu Glu Ile           
            570                 575                 580                   

ttc aac ccc gat atc ggc atg ttt acc tac gac gag tct aca aag ctg       2489
Phe Asn Pro Asp Ile Gly Met Phe Thr Tyr Asp Glu Ser Thr Lys Leu           
        585                 590                 595                       

ttc tgg ttt aat cct tct tcc ttc gag acc gag ggc cag ttt aca ctg       2537
Phe Trp Phe Asn Pro Ser Ser Phe Glu Thr Glu Gly Gln Phe Thr Leu           
    600                 605                 610                           

atc ggc atc gtg ctg ggc ctg gcc atc tac aac aat tgt atc ctg gac       2585
Ile Gly Ile Val Leu Gly Leu Ala Ile Tyr Asn Asn Cys Ile Leu Asp           
615                 620                 625                 630           

gtg cac ttc cca atg gtg gtg tat agg aag ctg atg ggc aag aag ggc       2633
Val His Phe Pro Met Val Val Tyr Arg Lys Leu Met Gly Lys Lys Gly           
                635                 640                 645               

acc ttt cgc gat ctg ggc gac agc cac ccc gtg ctg tac cag tcc ctg       2681
Thr Phe Arg Asp Leu Gly Asp Ser His Pro Val Leu Tyr Gln Ser Leu           
            650                 655                 660                   

aag gat ctg ctg gag tat gag ggc aac gtg gag gat gac atg atg atc       2729
Lys Asp Leu Leu Glu Tyr Glu Gly Asn Val Glu Asp Asp Met Met Ile           
        665                 670                 675                       

acc ttc cag atc tcc cag aca gac ctg ttt ggc aac cca atg atg tac       2777
Thr Phe Gln Ile Ser Gln Thr Asp Leu Phe Gly Asn Pro Met Met Tyr           
    680                 685                 690                           

gat ctg aag gag aac ggc gac aag atc ccc atc aca aac gag aat agg       2825
Asp Leu Lys Glu Asn Gly Asp Lys Ile Pro Ile Thr Asn Glu Asn Arg           
695                 700                 705                 710           

aag gag ttc gtg aac ctg tac tct gat tat atc ctg aat aag agc gtg       2873
Lys Glu Phe Val Asn Leu Tyr Ser Asp Tyr Ile Leu Asn Lys Ser Val           
                715                 720                 725               

gag aag cag ttc aag gcc ttt agg cgc ggc ttc cac atg gtg acc aac       2921
Glu Lys Gln Phe Lys Ala Phe Arg Arg Gly Phe His Met Val Thr Asn           
            730                 735                 740                   

gag tct cct ctg aag tat ctg ttt agg cca gag gag atc gag ctg ctg       2969
Glu Ser Pro Leu Lys Tyr Leu Phe Arg Pro Glu Glu Ile Glu Leu Leu           
        745                 750                 755                       

atc tgc ggc agc cgc aat ctg gac ttt cag gcc ctg gag gag acc aca       3017
Ile Cys Gly Ser Arg Asn Leu Asp Phe Gln Ala Leu Glu Glu Thr Thr           
    760                 765                 770                           

gag tac gac ggc ggc tat acc cgg gac tcc gtg ctg atc aga gag ttc       3065
Glu Tyr Asp Gly Gly Tyr Thr Arg Asp Ser Val Leu Ile Arg Glu Phe           
775                 780                 785                 790           

tgg gag atc gtg cac tct ttt aca gac gag cag aag cgg ctg ttc ctg       3113
Trp Glu Ile Val His Ser Phe Thr Asp Glu Gln Lys Arg Leu Phe Leu           
                795                 800                 805               

cag ttt acc acc ggc acc gac aga gca cca gtg gga gga ctg ggc aag       3161
Gln Phe Thr Thr Gly Thr Asp Arg Ala Pro Val Gly Gly Leu Gly Lys           
            810                 815                 820                   

ctg aag atg atc atc gcc aag aac ggc cca gac aca gag agg ctg ccc       3209
Leu Lys Met Ile Ile Ala Lys Asn Gly Pro Asp Thr Glu Arg Leu Pro           
        825                 830                 835                       

acc agc cac acc tgt ttc aac gtg ctg ctg ctg ccc gag tac tcc tct       3257
Thr Ser His Thr Cys Phe Asn Val Leu Leu Leu Pro Glu Tyr Ser Ser           
    840                 845                 850                           

aag gag aag ctg aag gag cgc ctg ctg aag gcc atc acc tac gcc aag       3305
Lys Glu Lys Leu Lys Glu Arg Leu Leu Lys Ala Ile Thr Tyr Ala Lys           
855                 860                 865                 870           

ggc ttt ggc atg ctg tga tga ggtaccagtg aattctacca gtgccatagg          3356
Gly Phe Gly Met Leu                                                       
                875                                                       

atagtgaatt ctaccagtgc catacacgtg agtgaattct accagtgcca tagcatgcag     3416

tgaattctac cagtgccata ggcggccgct tcgagcagac atgataagat acattgatga     3476

gtttggacaa accacaacta gaatgcagtg aaaaaaatgc tttatttgtg aaatttgtga     3536

tgctattgct ttatttgtaa ccattataag ctgcaataaa caagttaaca acaacaattg     3596

cattcatttt atgtttcagg ttcaggggga gatgtgggag gttttttaaa gcaagtaaaa     3656

cctctacaaa tgtggtaaaa tcgataagga tcttcctaga gcatggctac gtagataagt     3716

agcatggcgg gttaatcatt aactacaagg aacccctagt gatggagttg gccactccct     3776

ctctgcgcgc tcgctcgctc actgaggccg ggcgaccaaa ggtcgcccga cgcccgggct     3836

ttgcccgggc ggcctcagtg agcgagcgag cgcgcag                              3873


<210>  6
<211>  875
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Synthetic Construct

<400>  6

Met Glu Lys Leu His Gln Cys Tyr Trp Lys Ser Gly Glu Pro Gln Ser 
1               5                   10                  15      


Asp Asp Ile Glu Ala Ser Arg Met Lys Arg Ala Ala Ala Lys His Leu 
            20                  25                  30          


Ile Glu Arg Tyr Tyr His Gln Leu Thr Glu Gly Cys Gly Asn Glu Ala 
        35                  40                  45              


Cys Thr Asn Glu Phe Cys Ala Ser Cys Pro Thr Phe Leu Arg Met Asp 
    50                  55                  60                  


Asn Asn Ala Ala Ala Ile Lys Ala Leu Glu Leu Tyr Lys Ile Asn Ala 
65                  70                  75                  80  


Lys Leu Cys Asp Pro His Pro Ser Lys Lys Gly Ala Ser Ser Ala Tyr 
                85                  90                  95      


Leu Glu Asn Ser Lys Gly Ala Pro Asn Asn Ser Cys Ser Glu Ile Lys 
            100                 105                 110         


Met Asn Lys Lys Gly Ala Arg Ile Asp Phe Lys Asp Val Thr Tyr Leu 
        115                 120                 125             


Thr Glu Glu Lys Val Tyr Glu Ile Leu Glu Leu Cys Arg Glu Arg Glu 
    130                 135                 140                 


Asp Tyr Ser Pro Leu Ile Arg Val Ile Gly Arg Val Phe Ser Ser Ala 
145                 150                 155                 160 


Glu Ala Leu Val Gln Ser Phe Arg Lys Val Lys Gln His Thr Lys Glu 
                165                 170                 175     


Glu Leu Lys Ser Leu Gln Ala Lys Asp Glu Asp Lys Asp Glu Asp Glu 
            180                 185                 190         


Lys Glu Lys Ala Ala Cys Ser Ala Ala Ala Met Glu Glu Asp Ser Glu 
        195                 200                 205             


Ala Ser Ser Ser Arg Ile Gly Asp Ser Ser Gln Gly Asp Asn Asn Leu 
    210                 215                 220                 


Gln Lys Leu Gly Pro Asp Asp Val Ser Val Asp Ile Asp Ala Ile Arg 
225                 230                 235                 240 


Arg Val Tyr Thr Arg Leu Leu Ser Asn Glu Lys Ile Glu Thr Ala Phe 
                245                 250                 255     


Leu Asn Ala Leu Val Tyr Leu Ser Pro Asn Val Glu Cys Asp Leu Thr 
            260                 265                 270         


Tyr His Asn Val Tyr Ser Arg Asp Pro Asn Tyr Leu Asn Leu Phe Ile 
        275                 280                 285             


Ile Val Met Glu Asn Arg Asn Leu His Ser Pro Glu Tyr Leu Glu Met 
    290                 295                 300                 


Ala Leu Pro Leu Phe Cys Lys Ala Met Ser Lys Leu Pro Leu Ala Ala 
305                 310                 315                 320 


Gln Gly Lys Leu Ile Arg Leu Trp Ser Lys Tyr Asn Ala Asp Gln Ile 
                325                 330                 335     


Arg Arg Met Met Glu Thr Phe Gln Gln Leu Ile Thr Tyr Lys Val Ile 
            340                 345                 350         


Ser Asn Glu Phe Asn Ser Arg Asn Leu Val Asn Asp Asp Asp Ala Ile 
        355                 360                 365             


Val Ala Ala Ser Lys Cys Leu Lys Met Val Tyr Tyr Ala Asn Val Val 
    370                 375                 380                 


Gly Gly Glu Val Asp Thr Asn His Asn Glu Glu Asp Asp Glu Glu Pro 
385                 390                 395                 400 


Ile Pro Glu Ser Ser Glu Leu Thr Leu Gln Glu Leu Leu Gly Glu Glu 
                405                 410                 415     


Arg Arg Asn Lys Lys Gly Pro Arg Val Asp Pro Leu Glu Thr Glu Leu 
            420                 425                 430         


Gly Val Lys Thr Leu Asp Cys Arg Lys Pro Leu Ile Pro Phe Glu Glu 
        435                 440                 445             


Phe Ile Asn Glu Pro Leu Asn Glu Val Leu Glu Met Asp Lys Asp Tyr 
    450                 455                 460                 


Thr Phe Phe Lys Val Glu Thr Glu Asn Lys Phe Ser Phe Met Thr Cys 
465                 470                 475                 480 


Pro Phe Ile Leu Asn Ala Val Thr Lys Asn Leu Gly Leu Tyr Tyr Asp 
                485                 490                 495     


Asn Arg Ile Arg Met Tyr Ser Glu Arg Arg Ile Thr Val Leu Tyr Ser 
            500                 505                 510         


Leu Val Gln Gly Gln Gln Leu Asn Pro Tyr Leu Arg Leu Lys Val Arg 
        515                 520                 525             


Arg Asp His Ile Ile Asp Asp Ala Leu Val Arg Leu Glu Met Ile Ala 
    530                 535                 540                 


Met Glu Asn Pro Ala Asp Leu Lys Lys Gln Leu Tyr Val Glu Phe Glu 
545                 550                 555                 560 


Gly Glu Gln Gly Val Asp Glu Gly Gly Val Ser Lys Glu Phe Phe Gln 
                565                 570                 575     


Leu Val Val Glu Glu Ile Phe Asn Pro Asp Ile Gly Met Phe Thr Tyr 
            580                 585                 590         


Asp Glu Ser Thr Lys Leu Phe Trp Phe Asn Pro Ser Ser Phe Glu Thr 
        595                 600                 605             


Glu Gly Gln Phe Thr Leu Ile Gly Ile Val Leu Gly Leu Ala Ile Tyr 
    610                 615                 620                 


Asn Asn Cys Ile Leu Asp Val His Phe Pro Met Val Val Tyr Arg Lys 
625                 630                 635                 640 


Leu Met Gly Lys Lys Gly Thr Phe Arg Asp Leu Gly Asp Ser His Pro 
                645                 650                 655     


Val Leu Tyr Gln Ser Leu Lys Asp Leu Leu Glu Tyr Glu Gly Asn Val 
            660                 665                 670         


Glu Asp Asp Met Met Ile Thr Phe Gln Ile Ser Gln Thr Asp Leu Phe 
        675                 680                 685             


Gly Asn Pro Met Met Tyr Asp Leu Lys Glu Asn Gly Asp Lys Ile Pro 
    690                 695                 700                 


Ile Thr Asn Glu Asn Arg Lys Glu Phe Val Asn Leu Tyr Ser Asp Tyr 
705                 710                 715                 720 


Ile Leu Asn Lys Ser Val Glu Lys Gln Phe Lys Ala Phe Arg Arg Gly 
                725                 730                 735     


Phe His Met Val Thr Asn Glu Ser Pro Leu Lys Tyr Leu Phe Arg Pro 
            740                 745                 750         


Glu Glu Ile Glu Leu Leu Ile Cys Gly Ser Arg Asn Leu Asp Phe Gln 
        755                 760                 765             


Ala Leu Glu Glu Thr Thr Glu Tyr Asp Gly Gly Tyr Thr Arg Asp Ser 
    770                 775                 780                 


Val Leu Ile Arg Glu Phe Trp Glu Ile Val His Ser Phe Thr Asp Glu 
785                 790                 795                 800 


Gln Lys Arg Leu Phe Leu Gln Phe Thr Thr Gly Thr Asp Arg Ala Pro 
                805                 810                 815     


Val Gly Gly Leu Gly Lys Leu Lys Met Ile Ile Ala Lys Asn Gly Pro 
            820                 825                 830         


Asp Thr Glu Arg Leu Pro Thr Ser His Thr Cys Phe Asn Val Leu Leu 
        835                 840                 845             


Leu Pro Glu Tyr Ser Ser Lys Glu Lys Leu Lys Glu Arg Leu Leu Lys 
    850                 855                 860                 


Ala Ile Thr Tyr Ala Lys Gly Phe Gly Met Leu 
865                 870                 875 


<210>  7
<211>  3769
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  vector genome hSyn.hUbe3a-2.GSco.SV40


<220>
<221>  repeat_region
<222>  (1)..(130)
<223>  ITR

<220>
<221>  promoter
<222>  (213)..(678)
<223>  hSynapsin promoter

<220>
<221>  misc_feature
<222>  (690)..(695)
<223>  Kozak

<220>
<221>  CDS
<222>  (696)..(3326)
<223>  hUBE3a-2

<220>
<221>  misc_feature
<222>  (848)..(848)
<223>  mut

<220>
<221>  misc_feature
<222>  (893)..(893)
<223>  mut

<220>
<221>  misc_feature
<222>  (1097)..(1097)
<223>  mut

<220>
<221>  misc_feature
<222>  (1250)..(1250)
<223>  mut

<220>
<221>  misc_feature
<222>  (1262)..(1262)
<223>  mut

<220>
<221>  misc_feature
<222>  (1286)..(1286)
<223>  mut

<220>
<221>  misc_feature
<222>  (1352)..(1352)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (1784)..(1784)
<223>  mut

<220>
<221>  misc_feature
<222>  (1790)..(1790)
<223>  mut

<220>
<221>  misc_feature
<222>  (1835)..(1835)
<223>  mut

<220>
<221>  misc_feature
<222>  (1874)..(1874)
<223>  mut

<220>
<221>  misc_feature
<222>  (1883)..(1883)
<223>  mut

<220>
<221>  misc_feature
<222>  (2060)..(2060)
<223>  mut

<220>
<221>  misc_feature
<222>  (2132)..(2132)
<223>  mut

<220>
<221>  misc_feature
<222>  (2180)..(2180)
<223>  mut

<220>
<221>  misc_feature
<222>  (2363)..(2363)
<223>  mut

<220>
<221>  misc_feature
<222>  (2508)..(2509)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (2792)..(2792)
<223>  mut

<220>
<221>  misc_feature
<222>  (3026)..(3026)
<223>  mut

<220>
<221>  misc_feature
<222>  (3092)..(3092)
<223>  mut

<220>
<221>  misc_feature
<222>  (3125)..(3125)
<223>  mut SD/AS

<220>
<221>  misc_feature
<222>  (3221)..(3221)
<223>  mut

<220>
<221>  misc_feature
<222>  (3230)..(3230)
<223>  mut

<220>
<221>  misc_feature
<222>  (3299)..(3299)
<223>  mut

<220>
<221>  polyA_signal
<222>  (3344)..(3757)
<223>  SV40 polyA

<220>
<221>  repeat_region
<222>  (3640)..(3769)
<223>  ITR

<400>  7
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct tgtagttaat gattaacccg ccatgctact tatctacgta gccatgctct      180

aggaagatcc tctagaacta tagctagcat gcctgcagag ggccctgcgt atgagtgcaa      240

gtgggtttta ggaccaggat gaggcggggt gggggtgcct acctgacgac cgaccccgac      300

ccactggaca agcacccaac ccccattccc caaattgcgc atcccctatc agagaggggg      360

aggggaaaca ggatgcggcg aggcgcgtgc gcactgccag cttcagcacc gcggacagtg      420

ccttcgcccc cgcctggcgg cgcgcgccac cgccgcctca gcactgaagg cgcgctgacg      480

tcactcgccg gtcccccgca aactcccctt cccggccacc ttggtcgcgt ccgcgccgcc      540

gccggcccag ccggaccgca ccacgcgagg cgcgagatag gggggcacgg gcgcgaccat      600

ctgcgctgcg gcgccggcga ctcagcgctg cctcagtctg cggtgggcag cggaggagtc      660

gtgtcgtgcc tgagagcgca gtcgaattcg ccacc atg gag aag ctg cac cag         713
                                       Met Glu Lys Leu His Gln            
                                       1               5                  

tgc tac tgg aag tct ggc gag cct cag agc gac gat atc gag gca agc        761
Cys Tyr Trp Lys Ser Gly Glu Pro Gln Ser Asp Asp Ile Glu Ala Ser           
            10                  15                  20                    

agg atg aag aga gca gca gcc aag cac ctg atc gag cgg tac tat cac        809
Arg Met Lys Arg Ala Ala Ala Lys His Leu Ile Glu Arg Tyr Tyr His           
        25                  30                  35                        

cag ctg acc gag ggc tgc gga aac gag gcc tgt aca aac gag ttc tgc        857
Gln Leu Thr Glu Gly Cys Gly Asn Glu Ala Cys Thr Asn Glu Phe Cys           
    40                  45                  50                            

gcc tcc tgt cca acc ttt ctg aga atg gat aac aac gcc gcc gcc atc        905
Ala Ser Cys Pro Thr Phe Leu Arg Met Asp Asn Asn Ala Ala Ala Ile           
55                  60                  65                  70            

aag gcc ctg gag ctg tac aag atc aac gcc aag ctg tgc gac ccc cac        953
Lys Ala Leu Glu Leu Tyr Lys Ile Asn Ala Lys Leu Cys Asp Pro His           
                75                  80                  85                

cct tct aag aag ggc gcc agc tcc gcc tat ctg gag aac agc aag ggc       1001
Pro Ser Lys Lys Gly Ala Ser Ser Ala Tyr Leu Glu Asn Ser Lys Gly           
            90                  95                  100                   

gcc ccc aac aat agc tgt tcc gag atc aag atg aat aag aag ggc gcc       1049
Ala Pro Asn Asn Ser Cys Ser Glu Ile Lys Met Asn Lys Lys Gly Ala           
        105                 110                 115                       

agg atc gat ttc aag gac gtg acc tac ctg aca gag gag aag gtg tac       1097
Arg Ile Asp Phe Lys Asp Val Thr Tyr Leu Thr Glu Glu Lys Val Tyr           
    120                 125                 130                           

gag atc ctg gag ctg tgc cgg gag aga gag gat tac tcc cct ctg atc       1145
Glu Ile Leu Glu Leu Cys Arg Glu Arg Glu Asp Tyr Ser Pro Leu Ile           
135                 140                 145                 150           

aga gtg atc gga cgc gtg ttc tct agc gcc gag gcc ctg gtg cag agc       1193
Arg Val Ile Gly Arg Val Phe Ser Ser Ala Glu Ala Leu Val Gln Ser           
                155                 160                 165               

ttt cgc aag gtg aag cag cac aca aag gag gag ctg aag tcc ctg cag       1241
Phe Arg Lys Val Lys Gln His Thr Lys Glu Glu Leu Lys Ser Leu Gln           
            170                 175                 180                   

gcc aag gac gag gac aag gac gag gac gag aag gag aag gca gct tgt       1289
Ala Lys Asp Glu Asp Lys Asp Glu Asp Glu Lys Glu Lys Ala Ala Cys           
        185                 190                 195                       

tcc gcc gca gca atg gag gag gac tct gag gcc tcc tct agc agg atc       1337
Ser Ala Ala Ala Met Glu Glu Asp Ser Glu Ala Ser Ser Ser Arg Ile           
    200                 205                 210                           

ggc gat tcc tct caa ggc gac aac aat ctg cag aag ctg ggc ccc gac       1385
Gly Asp Ser Ser Gln Gly Asp Asn Asn Leu Gln Lys Leu Gly Pro Asp           
215                 220                 225                 230           

gac gtg agc gtg gat atc gac gcc atc cgg aga gtg tac aca cgc ctg       1433
Asp Val Ser Val Asp Ile Asp Ala Ile Arg Arg Val Tyr Thr Arg Leu           
                235                 240                 245               

ctg tct aac gag aag atc gag acc gcc ttc ctg aac gcc ctg gtg tat       1481
Leu Ser Asn Glu Lys Ile Glu Thr Ala Phe Leu Asn Ala Leu Val Tyr           
            250                 255                 260                   

ctg tct cct aat gtg gag tgc gat ctg acc tac cac aac gtg tac agc       1529
Leu Ser Pro Asn Val Glu Cys Asp Leu Thr Tyr His Asn Val Tyr Ser           
        265                 270                 275                       

cgg gac cca aac tac ctg aat ctg ttc atc atc gtg atg gag aac aga       1577
Arg Asp Pro Asn Tyr Leu Asn Leu Phe Ile Ile Val Met Glu Asn Arg           
    280                 285                 290                           

aat ctg cac tct ccc gag tat ctg gag atg gcc ctg cct ctg ttt tgt       1625
Asn Leu His Ser Pro Glu Tyr Leu Glu Met Ala Leu Pro Leu Phe Cys           
295                 300                 305                 310           

aag gcc atg agc aag ctg cca ctg gca gca cag ggc aag ctg atc agg       1673
Lys Ala Met Ser Lys Leu Pro Leu Ala Ala Gln Gly Lys Leu Ile Arg           
                315                 320                 325               

ctg tgg tcc aag tac aac gcc gat cag atc agg cgc atg atg gag acc       1721
Leu Trp Ser Lys Tyr Asn Ala Asp Gln Ile Arg Arg Met Met Glu Thr           
            330                 335                 340                   

ttc cag cag ctg atc aca tat aaa gtg atc tcc aac gag ttt aat tct       1769
Phe Gln Gln Leu Ile Thr Tyr Lys Val Ile Ser Asn Glu Phe Asn Ser           
        345                 350                 355                       

aga aac ctg gtg aac gac gac gac gcc atc gtg gcc gcc agc aag tgc       1817
Arg Asn Leu Val Asn Asp Asp Asp Ala Ile Val Ala Ala Ser Lys Cys           
    360                 365                 370                           

ctg aag atg gtg tac tac gcc aac gtg gtg ggc ggc gag gtg gac aca       1865
Leu Lys Met Val Tyr Tyr Ala Asn Val Val Gly Gly Glu Val Asp Thr           
375                 380                 385                 390           

aac cac aac gag gag gac gac gag gag cca atc ccc gag agc tcc gag       1913
Asn His Asn Glu Glu Asp Asp Glu Glu Pro Ile Pro Glu Ser Ser Glu           
                395                 400                 405               

ctg acc ctg cag gag ctg ctg gga gag gag cgg aga aat aag aag gga       1961
Leu Thr Leu Gln Glu Leu Leu Gly Glu Glu Arg Arg Asn Lys Lys Gly           
            410                 415                 420                   

cca agg gtg gat cct ctg gag acc gag ctg ggc gtg aag aca ctg gac       2009
Pro Arg Val Asp Pro Leu Glu Thr Glu Leu Gly Val Lys Thr Leu Asp           
        425                 430                 435                       

tgc aga aag cct ctg atc cca ttc gag gag ttt atc aac gag ccc ctg       2057
Cys Arg Lys Pro Leu Ile Pro Phe Glu Glu Phe Ile Asn Glu Pro Leu           
    440                 445                 450                           

aac gag gtg ctg gag atg gat aag gac tac acc ttc ttt aag gtg gag       2105
Asn Glu Val Leu Glu Met Asp Lys Asp Tyr Thr Phe Phe Lys Val Glu           
455                 460                 465                 470           

aca gag aac aag ttc agc ttt atg acc tgt cct ttc atc ctg aac gcc       2153
Thr Glu Asn Lys Phe Ser Phe Met Thr Cys Pro Phe Ile Leu Asn Ala           
                475                 480                 485               

gtg acc aag aat ctg ggc ctg tac tac gat aac agg atc cgc atg tac       2201
Val Thr Lys Asn Leu Gly Leu Tyr Tyr Asp Asn Arg Ile Arg Met Tyr           
            490                 495                 500                   

agc gag agg cgc atc acc gtg ctg tat tcc ctg gtg cag ggc cag cag       2249
Ser Glu Arg Arg Ile Thr Val Leu Tyr Ser Leu Val Gln Gly Gln Gln           
        505                 510                 515                       

ctg aat cct tac ctg agg ctg aag gtg cgg aga gac cac atc atc gat       2297
Leu Asn Pro Tyr Leu Arg Leu Lys Val Arg Arg Asp His Ile Ile Asp           
    520                 525                 530                           

gac gcc ctg gtg cgc ctg gag atg atc gcc atg gag aat cca gcc gat       2345
Asp Ala Leu Val Arg Leu Glu Met Ile Ala Met Glu Asn Pro Ala Asp           
535                 540                 545                 550           

ctg aag aag cag ctg tac gtg gag ttt gag gga gag cag gga gtg gac       2393
Leu Lys Lys Gln Leu Tyr Val Glu Phe Glu Gly Glu Gln Gly Val Asp           
                555                 560                 565               

gag gga ggc gtg tcc aag gag ttc ttt cag ctg gtg gtg gag gag atc       2441
Glu Gly Gly Val Ser Lys Glu Phe Phe Gln Leu Val Val Glu Glu Ile           
            570                 575                 580                   

ttc aac ccc gat atc ggc atg ttt acc tac gac gag tct aca aag ctg       2489
Phe Asn Pro Asp Ile Gly Met Phe Thr Tyr Asp Glu Ser Thr Lys Leu           
        585                 590                 595                       

ttc tgg ttt aat cct tct tcc ttc gag acc gag ggc cag ttt aca ctg       2537
Phe Trp Phe Asn Pro Ser Ser Phe Glu Thr Glu Gly Gln Phe Thr Leu           
    600                 605                 610                           

atc ggc atc gtg ctg ggc ctg gcc atc tac aac aat tgt atc ctg gac       2585
Ile Gly Ile Val Leu Gly Leu Ala Ile Tyr Asn Asn Cys Ile Leu Asp           
615                 620                 625                 630           

gtg cac ttc cca atg gtg gtg tat agg aag ctg atg ggc aag aag ggc       2633
Val His Phe Pro Met Val Val Tyr Arg Lys Leu Met Gly Lys Lys Gly           
                635                 640                 645               

acc ttt cgc gat ctg ggc gac agc cac ccc gtg ctg tac cag tcc ctg       2681
Thr Phe Arg Asp Leu Gly Asp Ser His Pro Val Leu Tyr Gln Ser Leu           
            650                 655                 660                   

aag gat ctg ctg gag tat gag ggc aac gtg gag gat gac atg atg atc       2729
Lys Asp Leu Leu Glu Tyr Glu Gly Asn Val Glu Asp Asp Met Met Ile           
        665                 670                 675                       

acc ttc cag atc tcc cag aca gac ctg ttt ggc aac cca atg atg tac       2777
Thr Phe Gln Ile Ser Gln Thr Asp Leu Phe Gly Asn Pro Met Met Tyr           
    680                 685                 690                           

gat ctg aag gag aac ggc gac aag atc ccc atc aca aac gag aat agg       2825
Asp Leu Lys Glu Asn Gly Asp Lys Ile Pro Ile Thr Asn Glu Asn Arg           
695                 700                 705                 710           

aag gag ttc gtg aac ctg tac tct gat tat atc ctg aat aag agc gtg       2873
Lys Glu Phe Val Asn Leu Tyr Ser Asp Tyr Ile Leu Asn Lys Ser Val           
                715                 720                 725               

gag aag cag ttc aag gcc ttt agg cgc ggc ttc cac atg gtg acc aac       2921
Glu Lys Gln Phe Lys Ala Phe Arg Arg Gly Phe His Met Val Thr Asn           
            730                 735                 740                   

gag tct cct ctg aag tat ctg ttt agg cca gag gag atc gag ctg ctg       2969
Glu Ser Pro Leu Lys Tyr Leu Phe Arg Pro Glu Glu Ile Glu Leu Leu           
        745                 750                 755                       

atc tgc ggc agc cgc aat ctg gac ttt cag gcc ctg gag gag acc aca       3017
Ile Cys Gly Ser Arg Asn Leu Asp Phe Gln Ala Leu Glu Glu Thr Thr           
    760                 765                 770                           

gag tac gac ggc ggc tat acc cgg gac tcc gtg ctg atc aga gag ttc       3065
Glu Tyr Asp Gly Gly Tyr Thr Arg Asp Ser Val Leu Ile Arg Glu Phe           
775                 780                 785                 790           

tgg gag atc gtg cac tct ttt aca gac gag cag aag cgg ctg ttc ctg       3113
Trp Glu Ile Val His Ser Phe Thr Asp Glu Gln Lys Arg Leu Phe Leu           
                795                 800                 805               

cag ttt acc acc ggc acc gac aga gca cca gtg gga gga ctg ggc aag       3161
Gln Phe Thr Thr Gly Thr Asp Arg Ala Pro Val Gly Gly Leu Gly Lys           
            810                 815                 820                   

ctg aag atg atc atc gcc aag aac ggc cca gac aca gag agg ctg ccc       3209
Leu Lys Met Ile Ile Ala Lys Asn Gly Pro Asp Thr Glu Arg Leu Pro           
        825                 830                 835                       

acc agc cac acc tgt ttc aac gtg ctg ctg ctg ccc gag tac tcc tct       3257
Thr Ser His Thr Cys Phe Asn Val Leu Leu Leu Pro Glu Tyr Ser Ser           
    840                 845                 850                           

aag gag aag ctg aag gag cgc ctg ctg aag gcc atc acc tac gcc aag       3305
Lys Glu Lys Leu Lys Glu Arg Leu Leu Lys Ala Ile Thr Tyr Ala Lys           
855                 860                 865                 870           

ggc ttt ggc atg ctg tga tga ggtaccggcg gccgcttcga gcagacatga          3356
Gly Phe Gly Met Leu                                                       
                875                                                       

taagatacat tgatgagttt ggacaaacca caactagaat gcagtgaaaa aaatgcttta     3416

tttgtgaaat ttgtgatgct attgctttat ttgtaaccat tataagctgc aataaacaag     3476

ttaacaacaa caattgcatt cattttatgt ttcaggttca gggggagatg tgggaggttt     3536

tttaaagcaa gtaaaacctc tacaaatgtg gtaaaatcga taaggatctt cctagagcat     3596

ggctacgtag ataagtagca tggcgggtta atcattaact acaaggaacc cctagtgatg     3656

gagttggcca ctccctctct gcgcgctcgc tcgctcactg aggccgggcg accaaaggtc     3716

gcccgacgcc cgggctttgc ccgggcggcc tcagtgagcg agcgagcgcg cag            3769


<210>  8
<211>  875
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Synthetic Construct

<400>  8

Met Glu Lys Leu His Gln Cys Tyr Trp Lys Ser Gly Glu Pro Gln Ser 
1               5                   10                  15      


Asp Asp Ile Glu Ala Ser Arg Met Lys Arg Ala Ala Ala Lys His Leu 
            20                  25                  30          


Ile Glu Arg Tyr Tyr His Gln Leu Thr Glu Gly Cys Gly Asn Glu Ala 
        35                  40                  45              


Cys Thr Asn Glu Phe Cys Ala Ser Cys Pro Thr Phe Leu Arg Met Asp 
    50                  55                  60                  


Asn Asn Ala Ala Ala Ile Lys Ala Leu Glu Leu Tyr Lys Ile Asn Ala 
65                  70                  75                  80  


Lys Leu Cys Asp Pro His Pro Ser Lys Lys Gly Ala Ser Ser Ala Tyr 
                85                  90                  95      


Leu Glu Asn Ser Lys Gly Ala Pro Asn Asn Ser Cys Ser Glu Ile Lys 
            100                 105                 110         


Met Asn Lys Lys Gly Ala Arg Ile Asp Phe Lys Asp Val Thr Tyr Leu 
        115                 120                 125             


Thr Glu Glu Lys Val Tyr Glu Ile Leu Glu Leu Cys Arg Glu Arg Glu 
    130                 135                 140                 


Asp Tyr Ser Pro Leu Ile Arg Val Ile Gly Arg Val Phe Ser Ser Ala 
145                 150                 155                 160 


Glu Ala Leu Val Gln Ser Phe Arg Lys Val Lys Gln His Thr Lys Glu 
                165                 170                 175     


Glu Leu Lys Ser Leu Gln Ala Lys Asp Glu Asp Lys Asp Glu Asp Glu 
            180                 185                 190         


Lys Glu Lys Ala Ala Cys Ser Ala Ala Ala Met Glu Glu Asp Ser Glu 
        195                 200                 205             


Ala Ser Ser Ser Arg Ile Gly Asp Ser Ser Gln Gly Asp Asn Asn Leu 
    210                 215                 220                 


Gln Lys Leu Gly Pro Asp Asp Val Ser Val Asp Ile Asp Ala Ile Arg 
225                 230                 235                 240 


Arg Val Tyr Thr Arg Leu Leu Ser Asn Glu Lys Ile Glu Thr Ala Phe 
                245                 250                 255     


Leu Asn Ala Leu Val Tyr Leu Ser Pro Asn Val Glu Cys Asp Leu Thr 
            260                 265                 270         


Tyr His Asn Val Tyr Ser Arg Asp Pro Asn Tyr Leu Asn Leu Phe Ile 
        275                 280                 285             


Ile Val Met Glu Asn Arg Asn Leu His Ser Pro Glu Tyr Leu Glu Met 
    290                 295                 300                 


Ala Leu Pro Leu Phe Cys Lys Ala Met Ser Lys Leu Pro Leu Ala Ala 
305                 310                 315                 320 


Gln Gly Lys Leu Ile Arg Leu Trp Ser Lys Tyr Asn Ala Asp Gln Ile 
                325                 330                 335     


Arg Arg Met Met Glu Thr Phe Gln Gln Leu Ile Thr Tyr Lys Val Ile 
            340                 345                 350         


Ser Asn Glu Phe Asn Ser Arg Asn Leu Val Asn Asp Asp Asp Ala Ile 
        355                 360                 365             


Val Ala Ala Ser Lys Cys Leu Lys Met Val Tyr Tyr Ala Asn Val Val 
    370                 375                 380                 


Gly Gly Glu Val Asp Thr Asn His Asn Glu Glu Asp Asp Glu Glu Pro 
385                 390                 395                 400 


Ile Pro Glu Ser Ser Glu Leu Thr Leu Gln Glu Leu Leu Gly Glu Glu 
                405                 410                 415     


Arg Arg Asn Lys Lys Gly Pro Arg Val Asp Pro Leu Glu Thr Glu Leu 
            420                 425                 430         


Gly Val Lys Thr Leu Asp Cys Arg Lys Pro Leu Ile Pro Phe Glu Glu 
        435                 440                 445             


Phe Ile Asn Glu Pro Leu Asn Glu Val Leu Glu Met Asp Lys Asp Tyr 
    450                 455                 460                 


Thr Phe Phe Lys Val Glu Thr Glu Asn Lys Phe Ser Phe Met Thr Cys 
465                 470                 475                 480 


Pro Phe Ile Leu Asn Ala Val Thr Lys Asn Leu Gly Leu Tyr Tyr Asp 
                485                 490                 495     


Asn Arg Ile Arg Met Tyr Ser Glu Arg Arg Ile Thr Val Leu Tyr Ser 
            500                 505                 510         


Leu Val Gln Gly Gln Gln Leu Asn Pro Tyr Leu Arg Leu Lys Val Arg 
        515                 520                 525             


Arg Asp His Ile Ile Asp Asp Ala Leu Val Arg Leu Glu Met Ile Ala 
    530                 535                 540                 


Met Glu Asn Pro Ala Asp Leu Lys Lys Gln Leu Tyr Val Glu Phe Glu 
545                 550                 555                 560 


Gly Glu Gln Gly Val Asp Glu Gly Gly Val Ser Lys Glu Phe Phe Gln 
                565                 570                 575     


Leu Val Val Glu Glu Ile Phe Asn Pro Asp Ile Gly Met Phe Thr Tyr 
            580                 585                 590         


Asp Glu Ser Thr Lys Leu Phe Trp Phe Asn Pro Ser Ser Phe Glu Thr 
        595                 600                 605             


Glu Gly Gln Phe Thr Leu Ile Gly Ile Val Leu Gly Leu Ala Ile Tyr 
    610                 615                 620                 


Asn Asn Cys Ile Leu Asp Val His Phe Pro Met Val Val Tyr Arg Lys 
625                 630                 635                 640 


Leu Met Gly Lys Lys Gly Thr Phe Arg Asp Leu Gly Asp Ser His Pro 
                645                 650                 655     


Val Leu Tyr Gln Ser Leu Lys Asp Leu Leu Glu Tyr Glu Gly Asn Val 
            660                 665                 670         


Glu Asp Asp Met Met Ile Thr Phe Gln Ile Ser Gln Thr Asp Leu Phe 
        675                 680                 685             


Gly Asn Pro Met Met Tyr Asp Leu Lys Glu Asn Gly Asp Lys Ile Pro 
    690                 695                 700                 


Ile Thr Asn Glu Asn Arg Lys Glu Phe Val Asn Leu Tyr Ser Asp Tyr 
705                 710                 715                 720 


Ile Leu Asn Lys Ser Val Glu Lys Gln Phe Lys Ala Phe Arg Arg Gly 
                725                 730                 735     


Phe His Met Val Thr Asn Glu Ser Pro Leu Lys Tyr Leu Phe Arg Pro 
            740                 745                 750         


Glu Glu Ile Glu Leu Leu Ile Cys Gly Ser Arg Asn Leu Asp Phe Gln 
        755                 760                 765             


Ala Leu Glu Glu Thr Thr Glu Tyr Asp Gly Gly Tyr Thr Arg Asp Ser 
    770                 775                 780                 


Val Leu Ile Arg Glu Phe Trp Glu Ile Val His Ser Phe Thr Asp Glu 
785                 790                 795                 800 


Gln Lys Arg Leu Phe Leu Gln Phe Thr Thr Gly Thr Asp Arg Ala Pro 
                805                 810                 815     


Val Gly Gly Leu Gly Lys Leu Lys Met Ile Ile Ala Lys Asn Gly Pro 
            820                 825                 830         


Asp Thr Glu Arg Leu Pro Thr Ser His Thr Cys Phe Asn Val Leu Leu 
        835                 840                 845             


Leu Pro Glu Tyr Ser Ser Lys Glu Lys Leu Lys Glu Arg Leu Leu Lys 
    850                 855                 860                 


Ala Ile Thr Tyr Ala Lys Gly Phe Gly Met Leu 
865                 870                 875 


<210>  9
<211>  2562
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  nucleic acid sequence hUbe31a-1

<400>  9
atgaagaggg cagcagcaaa gcacctgatc gagagatact atcaccagct gaccgaggga       60

tgcggaaacg aggcatgtac aaacgagttc tgcgcctcct gtcccacctt tctgaggatg      120

gataacaacg ccgccgccat caaggccctg gagctgtaca agatcaacgc caagctgtgc      180

gacccccacc ctagcaagaa gggcgccagc tccgcctatc tggagaactc caagggcgcc      240

cctaacaata gctgttccga gatcaagatg aataagaagg gcgcccggat cgatttcaag      300

gacgtgacct acctgacaga ggagaaggtg tacgagatcc tggagctgtg ccgggagaga      360

gaggattaca gcccactgat cagagtgatc ggcagagtgt tctctagcgc cgaggccctg      420

gtgcagtcct ttagaaaggt gaagcagcac acaaaggagg agctgaagtc tctgcaggcc      480

aaggacgagg acaaggacga ggacgagaag gagaaggcag cctgttctgc cgcagcaatg      540

gaggaggaca gcgaggcatc ctctagccgg atcggcgatt cctctcaagg cgacaacaat      600

ctgcagaagc tgggccccga cgacgtgagc gtggatatcg acgccatccg gagagtgtac      660

acaagactgc tgagcaacga gaagatcgag accgccttcc tgaacgccct ggtgtatctg      720

agccctaatg tggagtgcga tctgacctac cacaacgtgt actcccggga cccaaactac      780

ctgaatctgt tcatcatcgt gatggagaac agaaatctgc actcccccga gtatctggag      840

atggccctgc ctctgttttg taaggccatg tccaagctgc ctctggcagc acagggcaag      900

ctgatcaggc tgtggtctaa gtacaacgcc gatcagatca ggcgcatgat ggagaccttc      960

cagcagctga tcacatacaa agtgatctct aacgagttta atagccgcaa cctggtgaac     1020

gacgacgacg ccatcgtggc cgcctctaag tgcctgaaga tggtgtacta cgccaacgtg     1080

gtgggcggcg aggtggacac aaaccacaac gaggaggacg acgaggagcc aatccccgag     1140

agctccgagc tgaccctgca ggagctgctg ggagaggagc ggagaaataa gaagggacca     1200

agggtggatc ctctggagac cgagctgggc gtgaagacac tggactgcag aaagcctctg     1260

atcccattcg aggagtttat caacgagccc ctgaacgagg tgctggagat ggataaggac     1320

tacaccttct ttaaggtgga gacagagaac aagttcagct ttatgacctg tcctttcatc     1380

ctgaacgccg tgaccaagaa tctgggcctg tactacgata acaggatccg catgtactcc     1440

gagaggcgca tcaccgtgct gtattctctg gtgcagggcc agcagctgaa tccttacctg     1500

aggctgaagg tgcggagaga ccacatcatc gatgacgccc tggtgcgcct ggagatgatc     1560

gccatggaga atccagccga tctgaagaag cagctgtacg tggagtttga gggagagcag     1620

ggagtggacg agggaggcgt gtctaaggag ttctttcagc tggtggtgga ggagatcttc     1680

aaccccgata tcggcatgtt tacctacgac gagagcacaa agctgttctg gtttaatcct     1740

tcttccttcg agaccgaggg ccagtttaca ctgatcggca tcgtgctggg cctggccatc     1800

tacaacaatt gtatcctgga cgtgcacttc ccaatggtgg tgtataggaa gctgatgggc     1860

aagaagggca cctttcgcga tctgggcgac tcccaccccg tgctgtacca gtctctgaag     1920

gatctgctgg agtatgaggg caacgtggag gatgacatga tgatcacctt ccagatctcc     1980

cagacagacc tgtttggcaa cccaatgatg tacgatctga aggagaacgg cgacaagatc     2040

cccatcacaa acgagaatag aaaggagttc gtgaacctgt acagcgatta tatcctgaat     2100

aagtccgtgg agaagcagtt caaggccttt aggcgcggct tccacatggt gaccaacgag     2160

agccctctga agtatctgtt taggccagag gagatcgagc tgctgatctg cggctcccgc     2220

aatctggact ttcaggccct ggaggagacc acagagtacg acggcggcta taccagggac     2280

tctgtgctga tccgcgagtt ctgggagatc gtgcacagct ttacagacga gcagaagcgg     2340

ctgttcctgc agtttaccac cggcaccgac agagcaccag tgggaggact gggcaagctg     2400

aagatgatca tcgccaagaa cggcccagac acagagaggc tgcccaccag ccacacctgt     2460

ttcaacgtgc tgctgctgcc cgagtactcc tctaaggaga agctgaagga gcgcctgctg     2520

aaggccatca cctacgccaa gggctttggc atgctgtgat ga                        2562


<210>  10
<211>  2631
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  nucleic acid sequence hUbe3a-2

<400>  10
atggagaagc tgcaccagtg ctactggaag tctggcgagc ctcagagcga cgatatcgag       60

gcaagcagga tgaagagagc agcagccaag cacctgatcg agcggtacta tcaccagctg      120

accgagggct gcggaaacga ggcctgtaca aacgagttct gcgcctcctg tccaaccttt      180

ctgagaatgg ataacaacgc cgccgccatc aaggccctgg agctgtacaa gatcaacgcc      240

aagctgtgcg acccccaccc ttctaagaag ggcgccagct ccgcctatct ggagaacagc      300

aagggcgccc ccaacaatag ctgttccgag atcaagatga ataagaaggg cgccaggatc      360

gatttcaagg acgtgaccta cctgacagag gagaaggtgt acgagatcct ggagctgtgc      420

cgggagagag aggattactc ccctctgatc agagtgatcg gacgcgtgtt ctctagcgcc      480

gaggccctgg tgcagagctt tcgcaaggtg aagcagcaca caaaggagga gctgaagtcc      540

ctgcaggcca aggacgagga caaggacgag gacgagaagg agaaggcagc ttgttccgcc      600

gcagcaatgg aggaggactc tgaggcctcc tctagcagga tcggcgattc ctctcaaggc      660

gacaacaatc tgcagaagct gggccccgac gacgtgagcg tggatatcga cgccatccgg      720

agagtgtaca cacgcctgct gtctaacgag aagatcgaga ccgccttcct gaacgccctg      780

gtgtatctgt ctcctaatgt ggagtgcgat ctgacctacc acaacgtgta cagccgggac      840

ccaaactacc tgaatctgtt catcatcgtg atggagaaca gaaatctgca ctctcccgag      900

tatctggaga tggccctgcc tctgttttgt aaggccatga gcaagctgcc actggcagca      960

cagggcaagc tgatcaggct gtggtccaag tacaacgccg atcagatcag gcgcatgatg     1020

gagaccttcc agcagctgat cacatataaa gtgatctcca acgagtttaa ttctagaaac     1080

ctggtgaacg acgacgacgc catcgtggcc gccagcaagt gcctgaagat ggtgtactac     1140

gccaacgtgg tgggcggcga ggtggacaca aaccacaacg aggaggacga cgaggagcca     1200

atccccgaga gctccgagct gaccctgcag gagctgctgg gagaggagcg gagaaataag     1260

aagggaccaa gggtggatcc tctggagacc gagctgggcg tgaagacact ggactgcaga     1320

aagcctctga tcccattcga ggagtttatc aacgagcccc tgaacgaggt gctggagatg     1380

gataaggact acaccttctt taaggtggag acagagaaca agttcagctt tatgacctgt     1440

cctttcatcc tgaacgccgt gaccaagaat ctgggcctgt actacgataa caggatccgc     1500

atgtacagcg agaggcgcat caccgtgctg tattccctgg tgcagggcca gcagctgaat     1560

ccttacctga ggctgaaggt gcggagagac cacatcatcg atgacgccct ggtgcgcctg     1620

gagatgatcg ccatggagaa tccagccgat ctgaagaagc agctgtacgt ggagtttgag     1680

ggagagcagg gagtggacga gggaggcgtg tccaaggagt tctttcagct ggtggtggag     1740

gagatcttca accccgatat cggcatgttt acctacgacg agtctacaaa gctgttctgg     1800

tttaatcctt cttccttcga gaccgagggc cagtttacac tgatcggcat cgtgctgggc     1860

ctggccatct acaacaattg tatcctggac gtgcacttcc caatggtggt gtataggaag     1920

ctgatgggca agaagggcac ctttcgcgat ctgggcgaca gccaccccgt gctgtaccag     1980

tccctgaagg atctgctgga gtatgagggc aacgtggagg atgacatgat gatcaccttc     2040

cagatctccc agacagacct gtttggcaac ccaatgatgt acgatctgaa ggagaacggc     2100

gacaagatcc ccatcacaaa cgagaatagg aaggagttcg tgaacctgta ctctgattat     2160

atcctgaata agagcgtgga gaagcagttc aaggccttta ggcgcggctt ccacatggtg     2220

accaacgagt ctcctctgaa gtatctgttt aggccagagg agatcgagct gctgatctgc     2280

ggcagccgca atctggactt tcaggccctg gaggagacca cagagtacga cggcggctat     2340

acccgggact ccgtgctgat cagagagttc tgggagatcg tgcactcttt tacagacgag     2400

cagaagcggc tgttcctgca gtttaccacc ggcaccgaca gagcaccagt gggaggactg     2460

ggcaagctga agatgatcat cgccaagaac ggcccagaca cagagaggct gcccaccagc     2520

cacacctgtt tcaacgtgct gctgctgccc gagtactcct ctaaggagaa gctgaaggag     2580

cgcctgctga aggccatcac ctacgccaag ggctttggca tgctgtgatg a              2631


<210>  11
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  miRNA183 targeting sequence

<400>  11
agtgaattct accagtgcca ta                                                22


<210>  12
<211>  466
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hSynapsin promoter

<400>  12
ctgcagaggg ccctgcgtat gagtgcaagt gggttttagg accaggatga ggcggggtgg       60

gggtgcctac ctgacgaccg accccgaccc actggacaag cacccaaccc ccattcccca      120

aattgcgcat cccctatcag agagggggag gggaaacagg atgcggcgag gcgcgtgcgc      180

actgccagct tcagcaccgc ggacagtgcc ttcgcccccg cctggcggcg cgcgccaccg      240

ccgcctcagc actgaaggcg cgctgacgtc actcgccggt cccccgcaaa ctccccttcc      300

cggccacctt ggtcgcgtcc gcgccgccgc cggcccagcc ggaccgcacc acgcgaggcg      360

cgagataggg gggcacgggc gcgaccatct gcgctgcggc gccggcgact cagcgctgcc      420

tcagtctgcg gtgggcagcg gaggagtcgt gtcgtgcctg agagcg                     466


<210>  13
<211>  232
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SV40 polyA

<400>  13
cgagcagaca tgataagata cattgatgag tttggacaaa ccacaactag aatgcagtga       60

aaaaaatgct ttatttgtga aatttgtgat gctattgctt tatttgtaac cattataagc      120

tgcaataaac aagttaacaa caacaattgc attcatttta tgtttcaggt tcagggggag      180

atgtgggagg ttttttaaag caagtaaaac ctctacaaat gtggtaaaat cg              232


<210>  14
<211>  2211
<212>  DNA
<213>  adeno-associated virus hu68


<220>
<221>  CDS
<222>  (1)..(2211)
<223>  AAVhu68 Capsid

<400>  14
atg gct gcc gat ggt tat ctt cca gat tgg ctc gag gac aac ctc agt         48
Met Ala Ala Asp Gly Tyr Leu Pro Asp Trp Leu Glu Asp Asn Leu Ser           
1               5                   10                  15                

gaa ggc att cgc gag tgg tgg gct ttg aaa cct gga gcc cct caa ccc         96
Glu Gly Ile Arg Glu Trp Trp Ala Leu Lys Pro Gly Ala Pro Gln Pro           
            20                  25                  30                    

aag gca aat caa caa cat caa gac aac gct cgg ggt ctt gtg ctt ccg        144
Lys Ala Asn Gln Gln His Gln Asp Asn Ala Arg Gly Leu Val Leu Pro           
        35                  40                  45                        

ggt tac aaa tac ctt gga ccc ggc aac gga ctc gac aag ggg gag ccg        192
Gly Tyr Lys Tyr Leu Gly Pro Gly Asn Gly Leu Asp Lys Gly Glu Pro           
    50                  55                  60                            

gtc aac gaa gca gac gcg gcg gcc ctc gag cac gac aag gcc tac gac        240
Val Asn Glu Ala Asp Ala Ala Ala Leu Glu His Asp Lys Ala Tyr Asp           
65                  70                  75                  80            

cag cag ctc aag gcc gga gac aac ccg tac ctc aag tac aac cac gcc        288
Gln Gln Leu Lys Ala Gly Asp Asn Pro Tyr Leu Lys Tyr Asn His Ala           
                85                  90                  95                

gac gcc gag ttc cag gag cgg ctc aaa gaa gat acg tct ttt ggg ggc        336
Asp Ala Glu Phe Gln Glu Arg Leu Lys Glu Asp Thr Ser Phe Gly Gly           
            100                 105                 110                   

aac ctc ggg cga gca gtc ttc cag gcc aaa aag agg ctt ctt gaa cct        384
Asn Leu Gly Arg Ala Val Phe Gln Ala Lys Lys Arg Leu Leu Glu Pro           
        115                 120                 125                       

ctt ggt ctg gtt gag gaa gcg gct aag acg gct cct gga aag aag agg        432
Leu Gly Leu Val Glu Glu Ala Ala Lys Thr Ala Pro Gly Lys Lys Arg           
    130                 135                 140                           

cct gta gag cag tct cct cag gaa ccg gac tcc tcc gtg ggt att ggc        480
Pro Val Glu Gln Ser Pro Gln Glu Pro Asp Ser Ser Val Gly Ile Gly           
145                 150                 155                 160           

aaa tcg ggt gca cag ccc gct aaa aag aga ctc aat ttc ggt cag act        528
Lys Ser Gly Ala Gln Pro Ala Lys Lys Arg Leu Asn Phe Gly Gln Thr           
                165                 170                 175               

ggc gac aca gag tca gtc ccc gac cct caa cca atc gga gaa cct ccc        576
Gly Asp Thr Glu Ser Val Pro Asp Pro Gln Pro Ile Gly Glu Pro Pro           
            180                 185                 190                   

gca gcc ccc tca ggt gtg gga tct ctt aca atg gct tca ggt ggt ggc        624
Ala Ala Pro Ser Gly Val Gly Ser Leu Thr Met Ala Ser Gly Gly Gly           
        195                 200                 205                       

gca cca gtg gca gac aat aac gaa ggt gcc gat gga gtg ggt agt tcc        672
Ala Pro Val Ala Asp Asn Asn Glu Gly Ala Asp Gly Val Gly Ser Ser           
    210                 215                 220                           

tcg gga aat tgg cat tgc gat tcc caa tgg ctg ggg gac aga gtc atc        720
Ser Gly Asn Trp His Cys Asp Ser Gln Trp Leu Gly Asp Arg Val Ile           
225                 230                 235                 240           

acc acc agc acc cga acc tgg gcc ctg ccc acc tac aac aat cac ctc        768
Thr Thr Ser Thr Arg Thr Trp Ala Leu Pro Thr Tyr Asn Asn His Leu           
                245                 250                 255               

tac aag caa atc tcc aac agc aca tct gga gga tct tca aat gac aac        816
Tyr Lys Gln Ile Ser Asn Ser Thr Ser Gly Gly Ser Ser Asn Asp Asn           
            260                 265                 270                   

gcc tac ttc ggc tac agc acc ccc tgg ggg tat ttt gac ttc aac aga        864
Ala Tyr Phe Gly Tyr Ser Thr Pro Trp Gly Tyr Phe Asp Phe Asn Arg           
        275                 280                 285                       

ttc cac tgc cac ttc tca cca cgt gac tgg caa aga ctc atc aac aac        912
Phe His Cys His Phe Ser Pro Arg Asp Trp Gln Arg Leu Ile Asn Asn           
    290                 295                 300                           

aac tgg gga ttc cgg cct aag cga ctc aac ttc aag ctc ttc aac att        960
Asn Trp Gly Phe Arg Pro Lys Arg Leu Asn Phe Lys Leu Phe Asn Ile           
305                 310                 315                 320           

cag gtc aaa gag gtt acg gac aac aat gga gtc aag acc atc gct aat       1008
Gln Val Lys Glu Val Thr Asp Asn Asn Gly Val Lys Thr Ile Ala Asn           
                325                 330                 335               

aac ctt acc agc acg gtc cag gtc ttc acg gac tca gac tat cag ctc       1056
Asn Leu Thr Ser Thr Val Gln Val Phe Thr Asp Ser Asp Tyr Gln Leu           
            340                 345                 350                   

ccg tac gtg ctc ggg tcg gct cac gag ggc tgc ctc ccg ccg ttc cca       1104
Pro Tyr Val Leu Gly Ser Ala His Glu Gly Cys Leu Pro Pro Phe Pro           
        355                 360                 365                       

gcg gac gtt ttc atg att cct cag tac ggg tat cta acg ctt aat gat       1152
Ala Asp Val Phe Met Ile Pro Gln Tyr Gly Tyr Leu Thr Leu Asn Asp           
    370                 375                 380                           

gga agc caa gcc gtg ggt cgt tcg tcc ttt tac tgc ctg gaa tat ttc       1200
Gly Ser Gln Ala Val Gly Arg Ser Ser Phe Tyr Cys Leu Glu Tyr Phe           
385                 390                 395                 400           

ccg tcg caa atg cta aga acg ggt aac aac ttc cag ttc agc tac gag       1248
Pro Ser Gln Met Leu Arg Thr Gly Asn Asn Phe Gln Phe Ser Tyr Glu           
                405                 410                 415               

ttt gag aac gta cct ttc cat agc agc tat gct cac agc caa agc ctg       1296
Phe Glu Asn Val Pro Phe His Ser Ser Tyr Ala His Ser Gln Ser Leu           
            420                 425                 430                   

gac cga ctc atg aat cca ctc atc gac caa tac ttg tac tat ctc tca       1344
Asp Arg Leu Met Asn Pro Leu Ile Asp Gln Tyr Leu Tyr Tyr Leu Ser           
        435                 440                 445                       

aag act att aac ggt tct gga cag aat caa caa acg cta aaa ttc agt       1392
Lys Thr Ile Asn Gly Ser Gly Gln Asn Gln Gln Thr Leu Lys Phe Ser           
    450                 455                 460                           

gtg gcc gga ccc agc aac atg gct gtc cag gga aga aac tac ata cct       1440
Val Ala Gly Pro Ser Asn Met Ala Val Gln Gly Arg Asn Tyr Ile Pro           
465                 470                 475                 480           

gga ccc agc tac cga caa caa cgt gtc tca acc act gtg act caa aac       1488
Gly Pro Ser Tyr Arg Gln Gln Arg Val Ser Thr Thr Val Thr Gln Asn           
                485                 490                 495               

aac aac agc gaa ttt gct tgg cct gga gct tct tct tgg gct ctc aat       1536
Asn Asn Ser Glu Phe Ala Trp Pro Gly Ala Ser Ser Trp Ala Leu Asn           
            500                 505                 510                   

gga cgt aat agc ttg atg aat cct gga cct gct atg gcc agc cac aaa       1584
Gly Arg Asn Ser Leu Met Asn Pro Gly Pro Ala Met Ala Ser His Lys           
        515                 520                 525                       

gaa gga gag gac cgt ttc ttt cct ttg tct gga tct tta att ttt ggc       1632
Glu Gly Glu Asp Arg Phe Phe Pro Leu Ser Gly Ser Leu Ile Phe Gly           
    530                 535                 540                           

aaa caa gga act gga aga gac aac gtg gat gcg gac aaa gtc atg ata       1680
Lys Gln Gly Thr Gly Arg Asp Asn Val Asp Ala Asp Lys Val Met Ile           
545                 550                 555                 560           

acc aac gaa gaa gaa att aaa act acc aac cca gta gca acg gag tcc       1728
Thr Asn Glu Glu Glu Ile Lys Thr Thr Asn Pro Val Ala Thr Glu Ser           
                565                 570                 575               

tat gga caa gtg gcc aca aac cac cag agt gcc caa gca cag gcg cag       1776
Tyr Gly Gln Val Ala Thr Asn His Gln Ser Ala Gln Ala Gln Ala Gln           
            580                 585                 590                   

acc ggc tgg gtt caa aac caa gga ata ctt ccg ggt atg gtt tgg cag       1824
Thr Gly Trp Val Gln Asn Gln Gly Ile Leu Pro Gly Met Val Trp Gln           
        595                 600                 605                       

gac aga gat gtg tac ctg caa gga ccc att tgg gcc aaa att cct cac       1872
Asp Arg Asp Val Tyr Leu Gln Gly Pro Ile Trp Ala Lys Ile Pro His           
    610                 615                 620                           

acg gac ggc aac ttt cac cct tct ccg ctg atg gga ggg ttt gga atg       1920
Thr Asp Gly Asn Phe His Pro Ser Pro Leu Met Gly Gly Phe Gly Met           
625                 630                 635                 640           

aag cac ccg cct cct cag atc ctc atc aaa aac aca cct gta cct gcg       1968
Lys His Pro Pro Pro Gln Ile Leu Ile Lys Asn Thr Pro Val Pro Ala           
                645                 650                 655               

gat cct cca acg gct ttc aac aag gac aag ctg aac tct ttc atc acc       2016
Asp Pro Pro Thr Ala Phe Asn Lys Asp Lys Leu Asn Ser Phe Ile Thr           
            660                 665                 670                   

cag tat tct act ggc caa gtc agc gtg gag att gag tgg gag ctg cag       2064
Gln Tyr Ser Thr Gly Gln Val Ser Val Glu Ile Glu Trp Glu Leu Gln           
        675                 680                 685                       

aag gaa aac agc aag cgc tgg aac ccg gag atc cag tac act tcc aac       2112
Lys Glu Asn Ser Lys Arg Trp Asn Pro Glu Ile Gln Tyr Thr Ser Asn           
    690                 695                 700                           

tat tac aag tct aat aat gtt gaa ttt gct gtt aat act gaa ggt gtt       2160
Tyr Tyr Lys Ser Asn Asn Val Glu Phe Ala Val Asn Thr Glu Gly Val           
705                 710                 715                 720           

tat tct gaa ccc cgc ccc att ggc acc aga tac ctg act cgt aat ctg       2208
Tyr Ser Glu Pro Arg Pro Ile Gly Thr Arg Tyr Leu Thr Arg Asn Leu           
                725                 730                 735               

taa                                                                   2211


<210>  15
<211>  736
<212>  PRT
<213>  adeno-associated virus hu68

<400>  15

Met Ala Ala Asp Gly Tyr Leu Pro Asp Trp Leu Glu Asp Asn Leu Ser 
1               5                   10                  15      


Glu Gly Ile Arg Glu Trp Trp Ala Leu Lys Pro Gly Ala Pro Gln Pro 
            20                  25                  30          


Lys Ala Asn Gln Gln His Gln Asp Asn Ala Arg Gly Leu Val Leu Pro 
        35                  40                  45              


Gly Tyr Lys Tyr Leu Gly Pro Gly Asn Gly Leu Asp Lys Gly Glu Pro 
    50                  55                  60                  


Val Asn Glu Ala Asp Ala Ala Ala Leu Glu His Asp Lys Ala Tyr Asp 
65                  70                  75                  80  


Gln Gln Leu Lys Ala Gly Asp Asn Pro Tyr Leu Lys Tyr Asn His Ala 
                85                  90                  95      


Asp Ala Glu Phe Gln Glu Arg Leu Lys Glu Asp Thr Ser Phe Gly Gly 
            100                 105                 110         


Asn Leu Gly Arg Ala Val Phe Gln Ala Lys Lys Arg Leu Leu Glu Pro 
        115                 120                 125             


Leu Gly Leu Val Glu Glu Ala Ala Lys Thr Ala Pro Gly Lys Lys Arg 
    130                 135                 140                 


Pro Val Glu Gln Ser Pro Gln Glu Pro Asp Ser Ser Val Gly Ile Gly 
145                 150                 155                 160 


Lys Ser Gly Ala Gln Pro Ala Lys Lys Arg Leu Asn Phe Gly Gln Thr 
                165                 170                 175     


Gly Asp Thr Glu Ser Val Pro Asp Pro Gln Pro Ile Gly Glu Pro Pro 
            180                 185                 190         


Ala Ala Pro Ser Gly Val Gly Ser Leu Thr Met Ala Ser Gly Gly Gly 
        195                 200                 205             


Ala Pro Val Ala Asp Asn Asn Glu Gly Ala Asp Gly Val Gly Ser Ser 
    210                 215                 220                 


Ser Gly Asn Trp His Cys Asp Ser Gln Trp Leu Gly Asp Arg Val Ile 
225                 230                 235                 240 


Thr Thr Ser Thr Arg Thr Trp Ala Leu Pro Thr Tyr Asn Asn His Leu 
                245                 250                 255     


Tyr Lys Gln Ile Ser Asn Ser Thr Ser Gly Gly Ser Ser Asn Asp Asn 
            260                 265                 270         


Ala Tyr Phe Gly Tyr Ser Thr Pro Trp Gly Tyr Phe Asp Phe Asn Arg 
        275                 280                 285             


Phe His Cys His Phe Ser Pro Arg Asp Trp Gln Arg Leu Ile Asn Asn 
    290                 295                 300                 


Asn Trp Gly Phe Arg Pro Lys Arg Leu Asn Phe Lys Leu Phe Asn Ile 
305                 310                 315                 320 


Gln Val Lys Glu Val Thr Asp Asn Asn Gly Val Lys Thr Ile Ala Asn 
                325                 330                 335     


Asn Leu Thr Ser Thr Val Gln Val Phe Thr Asp Ser Asp Tyr Gln Leu 
            340                 345                 350         


Pro Tyr Val Leu Gly Ser Ala His Glu Gly Cys Leu Pro Pro Phe Pro 
        355                 360                 365             


Ala Asp Val Phe Met Ile Pro Gln Tyr Gly Tyr Leu Thr Leu Asn Asp 
    370                 375                 380                 


Gly Ser Gln Ala Val Gly Arg Ser Ser Phe Tyr Cys Leu Glu Tyr Phe 
385                 390                 395                 400 


Pro Ser Gln Met Leu Arg Thr Gly Asn Asn Phe Gln Phe Ser Tyr Glu 
                405                 410                 415     


Phe Glu Asn Val Pro Phe His Ser Ser Tyr Ala His Ser Gln Ser Leu 
            420                 425                 430         


Asp Arg Leu Met Asn Pro Leu Ile Asp Gln Tyr Leu Tyr Tyr Leu Ser 
        435                 440                 445             


Lys Thr Ile Asn Gly Ser Gly Gln Asn Gln Gln Thr Leu Lys Phe Ser 
    450                 455                 460                 


Val Ala Gly Pro Ser Asn Met Ala Val Gln Gly Arg Asn Tyr Ile Pro 
465                 470                 475                 480 


Gly Pro Ser Tyr Arg Gln Gln Arg Val Ser Thr Thr Val Thr Gln Asn 
                485                 490                 495     


Asn Asn Ser Glu Phe Ala Trp Pro Gly Ala Ser Ser Trp Ala Leu Asn 
            500                 505                 510         


Gly Arg Asn Ser Leu Met Asn Pro Gly Pro Ala Met Ala Ser His Lys 
        515                 520                 525             


Glu Gly Glu Asp Arg Phe Phe Pro Leu Ser Gly Ser Leu Ile Phe Gly 
    530                 535                 540                 


Lys Gln Gly Thr Gly Arg Asp Asn Val Asp Ala Asp Lys Val Met Ile 
545                 550                 555                 560 


Thr Asn Glu Glu Glu Ile Lys Thr Thr Asn Pro Val Ala Thr Glu Ser 
                565                 570                 575     


Tyr Gly Gln Val Ala Thr Asn His Gln Ser Ala Gln Ala Gln Ala Gln 
            580                 585                 590         


Thr Gly Trp Val Gln Asn Gln Gly Ile Leu Pro Gly Met Val Trp Gln 
        595                 600                 605             


Asp Arg Asp Val Tyr Leu Gln Gly Pro Ile Trp Ala Lys Ile Pro His 
    610                 615                 620                 


Thr Asp Gly Asn Phe His Pro Ser Pro Leu Met Gly Gly Phe Gly Met 
625                 630                 635                 640 


Lys His Pro Pro Pro Gln Ile Leu Ile Lys Asn Thr Pro Val Pro Ala 
                645                 650                 655     


Asp Pro Pro Thr Ala Phe Asn Lys Asp Lys Leu Asn Ser Phe Ile Thr 
            660                 665                 670         


Gln Tyr Ser Thr Gly Gln Val Ser Val Glu Ile Glu Trp Glu Leu Gln 
        675                 680                 685             


Lys Glu Asn Ser Lys Arg Trp Asn Pro Glu Ile Gln Tyr Thr Ser Asn 
    690                 695                 700                 


Tyr Tyr Lys Ser Asn Asn Val Glu Phe Ala Val Asn Thr Glu Gly Val 
705                 710                 715                 720 


Tyr Ser Glu Pro Arg Pro Ile Gly Thr Arg Tyr Leu Thr Arg Asn Leu 
                725                 730                 735     


<210>  16
<211>  2211
<212>  DNA
<213>  adeno-associated virus hu68

<400>  16
atggctgccg atggttatct tccagattgg ctcgaggaca acctcagtga aggcattcgc       60

gagtggtggg ctttgaaacc tggagcccct caacccaagg caaatcaaca acatcaagac      120

aacgctcggg gtcttgtgct tccgggttac aaataccttg gacccggcaa cggactcgac      180

aagggggagc cggtcaacga agcagacgcg gcggccctcg agcacgacaa ggcctacgac      240

cagcagctca aggccggaga caacccgtac ctcaagtaca accacgccga cgccgagttc      300

caggagcggc tcaaagaaga tacgtctttt gggggcaacc tcgggcgagc agtcttccag      360

gccaaaaaga ggcttcttga acctcttggt ctggttgagg aagcggctaa gacggctcct      420

ggaaagaaga ggcctgtaga gcagtctcct caggaaccgg actcctccgt gggtattggc      480

aaatcgggtg cacagcccgc taaaaagaga ctcaatttcg gtcagactgg cgacacagag      540

tcagtccccg accctcaacc aatcggagaa cctcccgcag ccccctcagg tgtgggatct      600

cttacaatgg cttcaggtgg tggcgcacca gtggcagaca ataacgaagg tgccgatgga      660

gtgggtagtt cctcgggaaa ttggcattgc gattcccaat ggctggggga cagagtcatc      720

accaccagca cccgaacctg ggccctgccc acctacaaca atcacctcta caagcaaatc      780

tccaacagca catctggagg atcttcaaat gacaacgcct acttcggcta cagcaccccc      840

tgggggtatt ttgacttcaa cagattccac tgccacttct caccacgtga ctggcaaaga      900

ctcatcaaca acaactgggg attccggcct aagcgactca acttcaagct cttcaacatt      960

caggtcaaag aggttacgga caacaatgga gtcaagacca tcgctaataa ccttaccagc     1020

acggtccagg tcttcacgga ctcagactat cagctcccgt acgtgctcgg gtcggctcac     1080

gagggctgcc tcccgccgtt cccagcggac gttttcatga ttcctcagta tggatacctc     1140

accctgaacg acggcagtca ggcggtgggc cgctcatcct tctactgcct ggagtacttc     1200

ccttcgcaga tgctgaggac tggcaacaac ttccagttca gctacgagtt cgagaacgtc     1260

cctttccaca gcagctacgc ccacagccag agtttggacc gcttgatgaa ccctctgatc     1320

gaccagtacc tgtactacct gtcaaagacg atcaacggtt ctggccagaa ccagcagacg     1380

ctgaagttca gcgtggccgg gcctagcaac atggccgtcc agggcagaaa ctacatccct     1440

gggcccagct accggcagca gagagtctca accactgtga ctcagaacaa caacagtgag     1500

ttcgcctggc ctggcgccag ctcttgggcc ctcaacggcc gcaactcgct gatgaaccca     1560

ggcccagcca tggccagtca caaggagggc gaggaccgtt tcttcccttt gtctggctct     1620

ctgatcttcg gcaagcaggg gaccggcaga gacaacgtgg acgcggacaa ggtcatgatc     1680

acgaacgagg aggagatcaa gaccaccaac cctgtggcaa ccgagtccta cggccaggtg     1740

gcaaccaacc accagagcgc ccaggcacag gcgcagactg gctgggtcca gaaccagggg     1800

atcctgcctg gcatggtgtg gcaggaccgt gacgtgtacc tgcagggccc tatctgggca     1860

aagatccctc acacggacgg caacttccac ccttctcctc tgatgggcgg cttcggcatg     1920

aagcacccgc ctcctcagat cctcatcaag aacactccgg tcccggcaga ccctccgacg     1980

gccttcaaca aggacaagct gaactcattc atcactcagt actccactgg ccaggtcagc     2040

gtggagatcg agtgggagct gcagaaggag aacagcaagc gttggaaccc agagatccag     2100

tacacttcca actactacaa gtctaacaac gtggagttcg ccgtcaacac tgagggtgtg     2160

tacagtgagc ctcgccctat cggcacccgg tacctcaccc gaaacttgtg a              2211


<210>  17
<211>  2211
<212>  DNA
<213>  adeno-associated virus rh.91


<220>
<221>  CDS
<222>  (1)..(2211)
<223>  AAVrh.91 capsid

<400>  17
atg gct gcc gat ggt tat ctt cca gat tgg ctc gag gac aac ctc tct         48
Met Ala Ala Asp Gly Tyr Leu Pro Asp Trp Leu Glu Asp Asn Leu Ser           
1               5                   10                  15                

gag ggc att cgc gag tgg tgg gcg ctg aaa cct gga gcc ccg aaa ccc         96
Glu Gly Ile Arg Glu Trp Trp Ala Leu Lys Pro Gly Ala Pro Lys Pro           
            20                  25                  30                    

aaa gcc aac cag caa aag cag gac gac ggc cgg ggt ctg gtg ctt cct        144
Lys Ala Asn Gln Gln Lys Gln Asp Asp Gly Arg Gly Leu Val Leu Pro           
        35                  40                  45                        

ggc tac aag tac ctc gga ccc ttc aac gga ctc gac aag ggg gag ccc        192
Gly Tyr Lys Tyr Leu Gly Pro Phe Asn Gly Leu Asp Lys Gly Glu Pro           
    50                  55                  60                            

gtc aac gcg gcg gac gca gcg gcc ctc gag cac gac aag gcc tac gac        240
Val Asn Ala Ala Asp Ala Ala Ala Leu Glu His Asp Lys Ala Tyr Asp           
65                  70                  75                  80            

cag cag ctc aaa gcg ggt gac aat ccg tac ctg cgg tat aac cac gcc        288
Gln Gln Leu Lys Ala Gly Asp Asn Pro Tyr Leu Arg Tyr Asn His Ala           
                85                  90                  95                

gac gcc gag ttt cag gag cgt ctg caa gaa gat acg tct ttt ggg ggc        336
Asp Ala Glu Phe Gln Glu Arg Leu Gln Glu Asp Thr Ser Phe Gly Gly           
            100                 105                 110                   

aac ctc ggg cga gca gtc ttc cag gcc aag aag cgg gtt ctc gaa cct        384
Asn Leu Gly Arg Ala Val Phe Gln Ala Lys Lys Arg Val Leu Glu Pro           
        115                 120                 125                       

ttt ggt ctg gtt gag gaa gca gct aag acg gct cct gga aag aaa cgt        432
Phe Gly Leu Val Glu Glu Ala Ala Lys Thr Ala Pro Gly Lys Lys Arg           
    130                 135                 140                           

ccg gta gag cag tcg ccc caa gaa cca gac tcc tcc tcg ggc att ggc        480
Pro Val Glu Gln Ser Pro Gln Glu Pro Asp Ser Ser Ser Gly Ile Gly           
145                 150                 155                 160           

aaa tca ggc cag cag ccc gcc aaa aag aga ctc aat ttc ggt cag act        528
Lys Ser Gly Gln Gln Pro Ala Lys Lys Arg Leu Asn Phe Gly Gln Thr           
                165                 170                 175               

ggc gac tca gag tca gtc ccc gac cct caa cct ctc gga gaa cct cca        576
Gly Asp Ser Glu Ser Val Pro Asp Pro Gln Pro Leu Gly Glu Pro Pro           
            180                 185                 190                   

gaa acc ccc gct gct gtg gga cct act aca atg gct tca ggc ggt ggc        624
Glu Thr Pro Ala Ala Val Gly Pro Thr Thr Met Ala Ser Gly Gly Gly           
        195                 200                 205                       

gca cca atg gca gac aat aac gaa ggc gcc gac gga gtg ggt aat gcc        672
Ala Pro Met Ala Asp Asn Asn Glu Gly Ala Asp Gly Val Gly Asn Ala           
    210                 215                 220                           

tca gga aat tgg cat tgc gat tcc aca tgg ctg ggc gac aga gtc atc        720
Ser Gly Asn Trp His Cys Asp Ser Thr Trp Leu Gly Asp Arg Val Ile           
225                 230                 235                 240           

acc acc agc acc cga acc tgg gcc ctt cct acc tac aac aac cac ctc        768
Thr Thr Ser Thr Arg Thr Trp Ala Leu Pro Thr Tyr Asn Asn His Leu           
                245                 250                 255               

tac aag caa atc tcc agc gct tca acg ggg gcc agt aac gac aac cac        816
Tyr Lys Gln Ile Ser Ser Ala Ser Thr Gly Ala Ser Asn Asp Asn His           
            260                 265                 270                   

tac ttt ggc tac agc acc ccc tgg ggg tat ttt gat ttc aac aga ttc        864
Tyr Phe Gly Tyr Ser Thr Pro Trp Gly Tyr Phe Asp Phe Asn Arg Phe           
        275                 280                 285                       

cac tgc cac ttc tca cca cgt gac tgg cag cga ctc att aac aac aac        912
His Cys His Phe Ser Pro Arg Asp Trp Gln Arg Leu Ile Asn Asn Asn           
    290                 295                 300                           

tgg gga ttc cgg ccc aag aga ctc aac ttc aag ctc ttc aac atc cag        960
Trp Gly Phe Arg Pro Lys Arg Leu Asn Phe Lys Leu Phe Asn Ile Gln           
305                 310                 315                 320           

gtc aag gag gtc acg acg aat gat ggc gtc aca acc atc gct aat aac       1008
Val Lys Glu Val Thr Thr Asn Asp Gly Val Thr Thr Ile Ala Asn Asn           
                325                 330                 335               

ctt acc agc acg gtt caa gtg ttc tcg gac tcg gag tac cag ctg ccg       1056
Leu Thr Ser Thr Val Gln Val Phe Ser Asp Ser Glu Tyr Gln Leu Pro           
            340                 345                 350                   

tac gtc ctc ggt tct gcg cac cag ggc tgc ctc cct ccg ttc ccg gcg       1104
Tyr Val Leu Gly Ser Ala His Gln Gly Cys Leu Pro Pro Phe Pro Ala           
        355                 360                 365                       

gac gta ttc atg att cct cag tac ggc tac cta acg ctc aac aat ggc       1152
Asp Val Phe Met Ile Pro Gln Tyr Gly Tyr Leu Thr Leu Asn Asn Gly           
    370                 375                 380                           

agc cag gcc gta gga cgt tca tcc ttt tat tgc ctg gaa tat ttc cca       1200
Ser Gln Ala Val Gly Arg Ser Ser Phe Tyr Cys Leu Glu Tyr Phe Pro           
385                 390                 395                 400           

tct caa atg ctg aga acg ggc aac aac ttt acc ttc agc tac acc ttt       1248
Ser Gln Met Leu Arg Thr Gly Asn Asn Phe Thr Phe Ser Tyr Thr Phe           
                405                 410                 415               

gaa gat gtg cct ttc cac agc agt tac gcg cac agc cag agc ctg gac       1296
Glu Asp Val Pro Phe His Ser Ser Tyr Ala His Ser Gln Ser Leu Asp           
            420                 425                 430                   

agg cta atg aat cct cta atc gac cag tac ctg tat tac cta aac aga       1344
Arg Leu Met Asn Pro Leu Ile Asp Gln Tyr Leu Tyr Tyr Leu Asn Arg           
        435                 440                 445                       

act cag aat caa tcc gga agt gca caa aac aag gac ttg ctg ttt agc       1392
Thr Gln Asn Gln Ser Gly Ser Ala Gln Asn Lys Asp Leu Leu Phe Ser           
    450                 455                 460                           

cgg ggg tct cca gct ggc atg tct gtt cag ccc aaa aac tgg cta ccc       1440
Arg Gly Ser Pro Ala Gly Met Ser Val Gln Pro Lys Asn Trp Leu Pro           
465                 470                 475                 480           

ggg ccc tgt tac cga cag cag cgt gtt tct aaa aca aaa aca gac aac       1488
Gly Pro Cys Tyr Arg Gln Gln Arg Val Ser Lys Thr Lys Thr Asp Asn           
                485                 490                 495               

aac aac agc aac ttt acc tgg act ggt gcc tcc aaa tac aat ctg aac       1536
Asn Asn Ser Asn Phe Thr Trp Thr Gly Ala Ser Lys Tyr Asn Leu Asn           
            500                 505                 510                   

gga cgt gaa tcc atc att aac cct ggc acc gct atg gca tcc cac aag       1584
Gly Arg Glu Ser Ile Ile Asn Pro Gly Thr Ala Met Ala Ser His Lys           
        515                 520                 525                       

gac gac gaa gac aaa ttt ttt ccc atg agc ggt gtt atg att ttt ggc       1632
Asp Asp Glu Asp Lys Phe Phe Pro Met Ser Gly Val Met Ile Phe Gly           
    530                 535                 540                           

aaa gaa aat gca gga gca tca aac act gca tta gac aat gtt atg att       1680
Lys Glu Asn Ala Gly Ala Ser Asn Thr Ala Leu Asp Asn Val Met Ile           
545                 550                 555                 560           

aca gat gaa gag gaa att aaa gct acc aac ccc gtg gcc acc gag aga       1728
Thr Asp Glu Glu Glu Ile Lys Ala Thr Asn Pro Val Ala Thr Glu Arg           
                565                 570                 575               

ttt gga act gtg gca gtc aat ctc caa agc agc aat aca gac cct gca       1776
Phe Gly Thr Val Ala Val Asn Leu Gln Ser Ser Asn Thr Asp Pro Ala           
            580                 585                 590                   

aca gga gac gtg cat gtc atg ggg gct tta cct ggc atg gtg tgg caa       1824
Thr Gly Asp Val His Val Met Gly Ala Leu Pro Gly Met Val Trp Gln           
        595                 600                 605                       

gac aga gac gtg tac ctg cag ggt ccc att tgg gcc aag att cct cac       1872
Asp Arg Asp Val Tyr Leu Gln Gly Pro Ile Trp Ala Lys Ile Pro His           
    610                 615                 620                           

acg gat gga cac ttt cac ccg tct cct ctt atg ggc ggc ttt gga ctt       1920
Thr Asp Gly His Phe His Pro Ser Pro Leu Met Gly Gly Phe Gly Leu           
625                 630                 635                 640           

aag cac ccg cct cct cag atc ctc atc aaa aac acg cct gtt cct gcg       1968
Lys His Pro Pro Pro Gln Ile Leu Ile Lys Asn Thr Pro Val Pro Ala           
                645                 650                 655               

aat cct ccg gca gag ttt tcg gct aca aag ttt gct tca ttc atc acc       2016
Asn Pro Pro Ala Glu Phe Ser Ala Thr Lys Phe Ala Ser Phe Ile Thr           
            660                 665                 670                   

cag tac tcc aca gga caa gtg agc gtg gaa att gaa tgg gag ctg cag       2064
Gln Tyr Ser Thr Gly Gln Val Ser Val Glu Ile Glu Trp Glu Leu Gln           
        675                 680                 685                       

aaa gaa aac agt aag cgc tgg aat cct gaa gtg cag tac acc tcc aac       2112
Lys Glu Asn Ser Lys Arg Trp Asn Pro Glu Val Gln Tyr Thr Ser Asn           
    690                 695                 700                           

tac gcg aaa tct gcc aac gtt gat ttc act gtg gac aac aat gga ctt       2160
Tyr Ala Lys Ser Ala Asn Val Asp Phe Thr Val Asp Asn Asn Gly Leu           
705                 710                 715                 720           

tat act gag cct cgc ccc att ggc acc cgt tac ctt acc cgt ccc ctt       2208
Tyr Thr Glu Pro Arg Pro Ile Gly Thr Arg Tyr Leu Thr Arg Pro Leu           
                725                 730                 735               

taa                                                                   2211


<210>  18
<211>  736
<212>  PRT
<213>  adeno-associated virus rh.91

<400>  18

Met Ala Ala Asp Gly Tyr Leu Pro Asp Trp Leu Glu Asp Asn Leu Ser 
1               5                   10                  15      


Glu Gly Ile Arg Glu Trp Trp Ala Leu Lys Pro Gly Ala Pro Lys Pro 
            20                  25                  30          


Lys Ala Asn Gln Gln Lys Gln Asp Asp Gly Arg Gly Leu Val Leu Pro 
        35                  40                  45              


Gly Tyr Lys Tyr Leu Gly Pro Phe Asn Gly Leu Asp Lys Gly Glu Pro 
    50                  55                  60                  


Val Asn Ala Ala Asp Ala Ala Ala Leu Glu His Asp Lys Ala Tyr Asp 
65                  70                  75                  80  


Gln Gln Leu Lys Ala Gly Asp Asn Pro Tyr Leu Arg Tyr Asn His Ala 
                85                  90                  95      


Asp Ala Glu Phe Gln Glu Arg Leu Gln Glu Asp Thr Ser Phe Gly Gly 
            100                 105                 110         


Asn Leu Gly Arg Ala Val Phe Gln Ala Lys Lys Arg Val Leu Glu Pro 
        115                 120                 125             


Phe Gly Leu Val Glu Glu Ala Ala Lys Thr Ala Pro Gly Lys Lys Arg 
    130                 135                 140                 


Pro Val Glu Gln Ser Pro Gln Glu Pro Asp Ser Ser Ser Gly Ile Gly 
145                 150                 155                 160 


Lys Ser Gly Gln Gln Pro Ala Lys Lys Arg Leu Asn Phe Gly Gln Thr 
                165                 170                 175     


Gly Asp Ser Glu Ser Val Pro Asp Pro Gln Pro Leu Gly Glu Pro Pro 
            180                 185                 190         


Glu Thr Pro Ala Ala Val Gly Pro Thr Thr Met Ala Ser Gly Gly Gly 
        195                 200                 205             


Ala Pro Met Ala Asp Asn Asn Glu Gly Ala Asp Gly Val Gly Asn Ala 
    210                 215                 220                 


Ser Gly Asn Trp His Cys Asp Ser Thr Trp Leu Gly Asp Arg Val Ile 
225                 230                 235                 240 


Thr Thr Ser Thr Arg Thr Trp Ala Leu Pro Thr Tyr Asn Asn His Leu 
                245                 250                 255     


Tyr Lys Gln Ile Ser Ser Ala Ser Thr Gly Ala Ser Asn Asp Asn His 
            260                 265                 270         


Tyr Phe Gly Tyr Ser Thr Pro Trp Gly Tyr Phe Asp Phe Asn Arg Phe 
        275                 280                 285             


His Cys His Phe Ser Pro Arg Asp Trp Gln Arg Leu Ile Asn Asn Asn 
    290                 295                 300                 


Trp Gly Phe Arg Pro Lys Arg Leu Asn Phe Lys Leu Phe Asn Ile Gln 
305                 310                 315                 320 


Val Lys Glu Val Thr Thr Asn Asp Gly Val Thr Thr Ile Ala Asn Asn 
                325                 330                 335     


Leu Thr Ser Thr Val Gln Val Phe Ser Asp Ser Glu Tyr Gln Leu Pro 
            340                 345                 350         


Tyr Val Leu Gly Ser Ala His Gln Gly Cys Leu Pro Pro Phe Pro Ala 
        355                 360                 365             


Asp Val Phe Met Ile Pro Gln Tyr Gly Tyr Leu Thr Leu Asn Asn Gly 
    370                 375                 380                 


Ser Gln Ala Val Gly Arg Ser Ser Phe Tyr Cys Leu Glu Tyr Phe Pro 
385                 390                 395                 400 


Ser Gln Met Leu Arg Thr Gly Asn Asn Phe Thr Phe Ser Tyr Thr Phe 
                405                 410                 415     


Glu Asp Val Pro Phe His Ser Ser Tyr Ala His Ser Gln Ser Leu Asp 
            420                 425                 430         


Arg Leu Met Asn Pro Leu Ile Asp Gln Tyr Leu Tyr Tyr Leu Asn Arg 
        435                 440                 445             


Thr Gln Asn Gln Ser Gly Ser Ala Gln Asn Lys Asp Leu Leu Phe Ser 
    450                 455                 460                 


Arg Gly Ser Pro Ala Gly Met Ser Val Gln Pro Lys Asn Trp Leu Pro 
465                 470                 475                 480 


Gly Pro Cys Tyr Arg Gln Gln Arg Val Ser Lys Thr Lys Thr Asp Asn 
                485                 490                 495     


Asn Asn Ser Asn Phe Thr Trp Thr Gly Ala Ser Lys Tyr Asn Leu Asn 
            500                 505                 510         


Gly Arg Glu Ser Ile Ile Asn Pro Gly Thr Ala Met Ala Ser His Lys 
        515                 520                 525             


Asp Asp Glu Asp Lys Phe Phe Pro Met Ser Gly Val Met Ile Phe Gly 
    530                 535                 540                 


Lys Glu Asn Ala Gly Ala Ser Asn Thr Ala Leu Asp Asn Val Met Ile 
545                 550                 555                 560 


Thr Asp Glu Glu Glu Ile Lys Ala Thr Asn Pro Val Ala Thr Glu Arg 
                565                 570                 575     


Phe Gly Thr Val Ala Val Asn Leu Gln Ser Ser Asn Thr Asp Pro Ala 
            580                 585                 590         


Thr Gly Asp Val His Val Met Gly Ala Leu Pro Gly Met Val Trp Gln 
        595                 600                 605             


Asp Arg Asp Val Tyr Leu Gln Gly Pro Ile Trp Ala Lys Ile Pro His 
    610                 615                 620                 


Thr Asp Gly His Phe His Pro Ser Pro Leu Met Gly Gly Phe Gly Leu 
625                 630                 635                 640 


Lys His Pro Pro Pro Gln Ile Leu Ile Lys Asn Thr Pro Val Pro Ala 
                645                 650                 655     


Asn Pro Pro Ala Glu Phe Ser Ala Thr Lys Phe Ala Ser Phe Ile Thr 
            660                 665                 670         


Gln Tyr Ser Thr Gly Gln Val Ser Val Glu Ile Glu Trp Glu Leu Gln 
        675                 680                 685             


Lys Glu Asn Ser Lys Arg Trp Asn Pro Glu Val Gln Tyr Thr Ser Asn 
    690                 695                 700                 


Tyr Ala Lys Ser Ala Asn Val Asp Phe Thr Val Asp Asn Asn Gly Leu 
705                 710                 715                 720 


Tyr Thr Glu Pro Arg Pro Ile Gly Thr Arg Tyr Leu Thr Arg Pro Leu 
                725                 730                 735     


<210>  19
<211>  2211
<212>  DNA
<213>  adeno-associated virus rh.91

<400>  19
atggctgctg acggttatct tccagattgg ctcgaggaca acctttctga aggcattcgt       60

gagtggtggg ctctgaaacc tggagcccct aaacccaaag cgaaccaaca aaagcaggac      120

gacggccggg gtcttgtgct tccgggttac aaatacctcg gacccttcaa cggactcgac      180

aaaggagagc cggtcaacgc ggcggacgcg gcagccctcg aacacgacaa agcttacgac      240

cagcagctca aggccggtga caacccgtac ctccggtaca accacgccga cgccgagttt      300

caggagcgtc ttcaagaaga tacgtctttt gggggcaacc ttggcagagc agtcttccag      360

gccaaaaaga gggttcttga gccttttggt ctggttgagg aagcagctaa aacggctcct      420

ggaaagaaga ggcctgtaga gcagtctcct caggaaccgg actcatcatc tggtattggc      480

aaatcgggcc agcagcctgc caaaaaaaga ctaaatttcg gtcagactgg cgactcagag      540

tcagtccccg accctcaacc tctcggagaa cctccagaaa cccccgctgc tgtgggacct      600

actacaatgg cttcaggcgg tggcgcacca atggcagaca ataacgaagg cgccgacgga      660

gtgggtaatg cctcaggaaa ttggcattgc gattccacat ggctgggcga cagagtcatc      720

accaccagca cccgaacctg ggcccttcct acctacaaca accacctcta caagcaaatc      780

tccagcgctt caacgggggc cagtaacgac aaccactact ttggctacag caccccctgg      840

gggtattttg atttcaacag attccactgc cacttctcac cacgtgactg gcagcgactc      900

attaacaaca actggggatt ccggcccaag agactcaact tcaagctctt caacatccag      960

gtcaaggagg tcacgacgaa tgatggcgtc acaaccatcg ctaataacct taccagcacg     1020

gttcaagtgt tctcggactc ggagtaccag ctgccgtacg tcctcggttc tgcgcaccag     1080

ggctgcctcc ctccgttccc ggcggacgta ttcatgattc ctcagtatgg atacctcacc     1140

ctgaacaacg gaagtcaagc ggtgggacgc tcatcctttt actgcctgga gtacttccct     1200

tcgcagatgc taaggactgg aaataacttc accttcagct ataccttcga ggatgtacct     1260

tttcacagca gctacgctca cagccagagt ttggatcgct tgatgaatcc tcttattgat     1320

cagtatctgt actacctgaa cagaacgcaa aatcaatctg gaagtgcaca aaacaaggac     1380

ctgcttttta gccgggggtc tcctgctggc atgtctgttc agcccaaaaa ttggctacct     1440

gggccctgct accggcaaca gagagtttca aagactaaaa cagacaacaa caacagtaac     1500

tttacctgga caggtgccag caaatataat ctcaatggcc gcgaatcgat cattaatcca     1560

ggaaccgcta tggccagtca caaggacgat gaagacaaat ttttccctat gagcggcgtt     1620

atgatatttg gcaaagaaaa tgcaggagca agtaacactg cattagataa tgtaatgatt     1680

acggatgaag aagagattaa agctaccaat cctgtggcaa cagagagatt tggaactgtg     1740

gcagtcaact tgcagagctc aaatacagac cccgcaactg gagacgtcca tgtcatgggg     1800

gccttacctg gcatggtgtg gcaagatcgt gacgtgtacc ttcaaggacc tatctgggca     1860

aagattcctc acacggatgg acactttcat ccttctcctc tgatgggagg ctttggactg     1920

aaacatccgc ctcctcaaat cctcatcaaa aatactccgg taccggcaaa tcctccggca     1980

gagttcagcg ctacaaagtt tgcttcattt atcactcagt actccactgg acaggtcagc     2040

gtggaaattg agtgggagct acagaaagaa aacagcaaac gttggaatcc agaggtgcag     2100

tacacttcca actacgcgaa gtctgccaat gtggacttta ctgtagacaa caatggtctt     2160

tatactgaac ctcgccctat tggaacccgg tatctcacac gacccttgta a              2211


<210>  20
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  miRNA182 targeting sequence

<400>  20
agtgtgagtt ctaccattgc caaa                                              24


<210>  21
<211>  872
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  hUBE3a isoform 3 amino acid sequence

<400>  21

Met Ala Thr Ala Cys Lys Arg Ser Gly Glu Pro Gln Ser Asp Asp Ile 
1               5                   10                  15      


Glu Ala Ser Arg Met Lys Arg Ala Ala Ala Lys His Leu Ile Glu Arg 
            20                  25                  30          


Tyr Tyr His Gln Leu Thr Glu Gly Cys Gly Asn Glu Ala Cys Thr Asn 
        35                  40                  45              


Glu Phe Cys Ala Ser Cys Pro Thr Phe Leu Arg Met Asp Asn Asn Ala 
    50                  55                  60                  


Ala Ala Ile Lys Ala Leu Glu Leu Tyr Lys Ile Asn Ala Lys Leu Cys 
65                  70                  75                  80  


Asp Pro His Pro Ser Lys Lys Gly Ala Ser Ser Ala Tyr Leu Glu Asn 
                85                  90                  95      


Ser Lys Gly Ala Pro Asn Asn Ser Cys Ser Glu Ile Lys Met Asn Lys 
            100                 105                 110         


Lys Gly Ala Arg Ile Asp Phe Lys Asp Val Thr Tyr Leu Thr Glu Glu 
        115                 120                 125             


Lys Val Tyr Glu Ile Leu Glu Leu Cys Arg Glu Arg Glu Asp Tyr Ser 
    130                 135                 140                 


Pro Leu Ile Arg Val Ile Gly Arg Val Phe Ser Ser Ala Glu Ala Leu 
145                 150                 155                 160 


Val Gln Ser Phe Arg Lys Val Lys Gln His Thr Lys Glu Glu Leu Lys 
                165                 170                 175     


Ser Leu Gln Ala Lys Asp Glu Asp Lys Asp Glu Asp Glu Lys Glu Lys 
            180                 185                 190         


Ala Ala Cys Ser Ala Ala Ala Met Glu Glu Asp Ser Glu Ala Ser Ser 
        195                 200                 205             


Ser Arg Ile Gly Asp Ser Ser Gln Gly Asp Asn Asn Leu Gln Lys Leu 
    210                 215                 220                 


Gly Pro Asp Asp Val Ser Val Asp Ile Asp Ala Ile Arg Arg Val Tyr 
225                 230                 235                 240 


Thr Arg Leu Leu Ser Asn Glu Lys Ile Glu Thr Ala Phe Leu Asn Ala 
                245                 250                 255     


Leu Val Tyr Leu Ser Pro Asn Val Glu Cys Asp Leu Thr Tyr His Asn 
            260                 265                 270         


Val Tyr Ser Arg Asp Pro Asn Tyr Leu Asn Leu Phe Ile Ile Val Met 
        275                 280                 285             


Glu Asn Arg Asn Leu His Ser Pro Glu Tyr Leu Glu Met Ala Leu Pro 
    290                 295                 300                 


Leu Phe Cys Lys Ala Met Ser Lys Leu Pro Leu Ala Ala Gln Gly Lys 
305                 310                 315                 320 


Leu Ile Arg Leu Trp Ser Lys Tyr Asn Ala Asp Gln Ile Arg Arg Met 
                325                 330                 335     


Met Glu Thr Phe Gln Gln Leu Ile Thr Tyr Lys Val Ile Ser Asn Glu 
            340                 345                 350         


Phe Asn Ser Arg Asn Leu Val Asn Asp Asp Asp Ala Ile Val Ala Ala 
        355                 360                 365             


Ser Lys Cys Leu Lys Met Val Tyr Tyr Ala Asn Val Val Gly Gly Glu 
    370                 375                 380                 


Val Asp Thr Asn His Asn Glu Glu Asp Asp Glu Glu Pro Ile Pro Glu 
385                 390                 395                 400 


Ser Ser Glu Leu Thr Leu Gln Glu Leu Leu Gly Glu Glu Arg Arg Asn 
                405                 410                 415     


Lys Lys Gly Pro Arg Val Asp Pro Leu Glu Thr Glu Leu Gly Val Lys 
            420                 425                 430         


Thr Leu Asp Cys Arg Lys Pro Leu Ile Pro Phe Glu Glu Phe Ile Asn 
        435                 440                 445             


Glu Pro Leu Asn Glu Val Leu Glu Met Asp Lys Asp Tyr Thr Phe Phe 
    450                 455                 460                 


Lys Val Glu Thr Glu Asn Lys Phe Ser Phe Met Thr Cys Pro Phe Ile 
465                 470                 475                 480 


Leu Asn Ala Val Thr Lys Asn Leu Gly Leu Tyr Tyr Asp Asn Arg Ile 
                485                 490                 495     


Arg Met Tyr Ser Glu Arg Arg Ile Thr Val Leu Tyr Ser Leu Val Gln 
            500                 505                 510         


Gly Gln Gln Leu Asn Pro Tyr Leu Arg Leu Lys Val Arg Arg Asp His 
        515                 520                 525             


Ile Ile Asp Asp Ala Leu Val Arg Leu Glu Met Ile Ala Met Glu Asn 
    530                 535                 540                 


Pro Ala Asp Leu Lys Lys Gln Leu Tyr Val Glu Phe Glu Gly Glu Gln 
545                 550                 555                 560 


Gly Val Asp Glu Gly Gly Val Ser Lys Glu Phe Phe Gln Leu Val Val 
                565                 570                 575     


Glu Glu Ile Phe Asn Pro Asp Ile Gly Met Phe Thr Tyr Asp Glu Ser 
            580                 585                 590         


Thr Lys Leu Phe Trp Phe Asn Pro Ser Ser Phe Glu Thr Glu Gly Gln 
        595                 600                 605             


Phe Thr Leu Ile Gly Ile Val Leu Gly Leu Ala Ile Tyr Asn Asn Cys 
    610                 615                 620                 


Ile Leu Asp Val His Phe Pro Met Val Val Tyr Arg Lys Leu Met Gly 
625                 630                 635                 640 


Lys Lys Gly Thr Phe Arg Asp Leu Gly Asp Ser His Pro Val Leu Tyr 
                645                 650                 655     


Gln Ser Leu Lys Asp Leu Leu Glu Tyr Glu Gly Asn Val Glu Asp Asp 
            660                 665                 670         


Met Met Ile Thr Phe Gln Ile Ser Gln Thr Asp Leu Phe Gly Asn Pro 
        675                 680                 685             


Met Met Tyr Asp Leu Lys Glu Asn Gly Asp Lys Ile Pro Ile Thr Asn 
    690                 695                 700                 


Glu Asn Arg Lys Glu Phe Val Asn Leu Tyr Ser Asp Tyr Ile Leu Asn 
705                 710                 715                 720 


Lys Ser Val Glu Lys Gln Phe Lys Ala Phe Arg Arg Gly Phe His Met 
                725                 730                 735     


Val Thr Asn Glu Ser Pro Leu Lys Tyr Leu Phe Arg Pro Glu Glu Ile 
            740                 745                 750         


Glu Leu Leu Ile Cys Gly Ser Arg Asn Leu Asp Phe Gln Ala Leu Glu 
        755                 760                 765             


Glu Thr Thr Glu Tyr Asp Gly Gly Tyr Thr Arg Asp Ser Val Leu Ile 
    770                 775                 780                 


Arg Glu Phe Trp Glu Ile Val His Ser Phe Thr Asp Glu Gln Lys Arg 
785                 790                 795                 800 


Leu Phe Leu Gln Phe Thr Thr Gly Thr Asp Arg Ala Pro Val Gly Gly 
                805                 810                 815     


Leu Gly Lys Leu Lys Met Ile Ile Ala Lys Asn Gly Pro Asp Thr Glu 
            820                 825                 830         


Arg Leu Pro Thr Ser His Thr Cys Phe Asn Val Leu Leu Leu Pro Glu 
        835                 840                 845             


Tyr Ser Ser Lys Glu Lys Leu Lys Glu Arg Leu Leu Lys Ala Ile Thr 
    850                 855                 860                 


Tyr Ala Lys Gly Phe Gly Met Leu 
865                 870         


<210>  22
<211>  3398
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  expression cassette hSyn.hUbe3a-1.GSco.4XmiRNA183.SV40


<220>
<221>  promoter
<222>  (1)..(466)
<223>  hSynapsin

<220>
<221>  misc_feature
<222>  (478)..(483)
<223>  Kozak

<220>
<221>  misc_feature
<222>  (484)..(3045)
<223>  UBE3A-1

<220>
<221>  misc_feature
<222>  (3052)..(3073)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3078)..(3099)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3106)..(3127)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3134)..(3155)
<223>  miRNA183

<220>
<221>  polyA_signal
<222>  (3167)..(3398)
<223>  SV40 late polyA

<400>  22
ctgcagaggg ccctgcgtat gagtgcaagt gggttttagg accaggatga ggcggggtgg       60

gggtgcctac ctgacgaccg accccgaccc actggacaag cacccaaccc ccattcccca      120

aattgcgcat cccctatcag agagggggag gggaaacagg atgcggcgag gcgcgtgcgc      180

actgccagct tcagcaccgc ggacagtgcc ttcgcccccg cctggcggcg cgcgccaccg      240

ccgcctcagc actgaaggcg cgctgacgtc actcgccggt cccccgcaaa ctccccttcc      300

cggccacctt ggtcgcgtcc gcgccgccgc cggcccagcc ggaccgcacc acgcgaggcg      360

cgagataggg gggcacgggc gcgaccatct gcgctgcggc gccggcgact cagcgctgcc      420

tcagtctgcg gtgggcagcg gaggagtcgt gtcgtgcctg agagcgcagt cgaattcgcc      480

accatgaaga gggcagcagc aaagcacctg atcgagagat actatcacca gctgaccgag      540

ggatgcggaa acgaggcatg tacaaacgag ttctgcgcct cctgtcccac ctttctgagg      600

atggataaca acgccgccgc catcaaggcc ctggagctgt acaagatcaa cgccaagctg      660

tgcgaccccc accctagcaa gaagggcgcc agctccgcct atctggagaa ctccaagggc      720

gcccctaaca atagctgttc cgagatcaag atgaataaga agggcgcccg gatcgatttc      780

aaggacgtga cctacctgac agaggagaag gtgtacgaga tcctggagct gtgccgggag      840

agagaggatt acagcccact gatcagagtg atcggcagag tgttctctag cgccgaggcc      900

ctggtgcagt cctttagaaa ggtgaagcag cacacaaagg aggagctgaa gtctctgcag      960

gccaaggacg aggacaagga cgaggacgag aaggagaagg cagcctgttc tgccgcagca     1020

atggaggagg acagcgaggc atcctctagc cggatcggcg attcctctca aggcgacaac     1080

aatctgcaga agctgggccc cgacgacgtg agcgtggata tcgacgccat ccggagagtg     1140

tacacaagac tgctgagcaa cgagaagatc gagaccgcct tcctgaacgc cctggtgtat     1200

ctgagcccta atgtggagtg cgatctgacc taccacaacg tgtactcccg ggacccaaac     1260

tacctgaatc tgttcatcat cgtgatggag aacagaaatc tgcactcccc cgagtatctg     1320

gagatggccc tgcctctgtt ttgtaaggcc atgtccaagc tgcctctggc agcacagggc     1380

aagctgatca ggctgtggtc taagtacaac gccgatcaga tcaggcgcat gatggagacc     1440

ttccagcagc tgatcacata caaagtgatc tctaacgagt ttaatagccg caacctggtg     1500

aacgacgacg acgccatcgt ggccgcctct aagtgcctga agatggtgta ctacgccaac     1560

gtggtgggcg gcgaggtgga cacaaaccac aacgaggagg acgacgagga gccaatcccc     1620

gagagctccg agctgaccct gcaggagctg ctgggagagg agcggagaaa taagaaggga     1680

ccaagggtgg atcctctgga gaccgagctg ggcgtgaaga cactggactg cagaaagcct     1740

ctgatcccat tcgaggagtt tatcaacgag cccctgaacg aggtgctgga gatggataag     1800

gactacacct tctttaaggt ggagacagag aacaagttca gctttatgac ctgtcctttc     1860

atcctgaacg ccgtgaccaa gaatctgggc ctgtactacg ataacaggat ccgcatgtac     1920

tccgagaggc gcatcaccgt gctgtattct ctggtgcagg gccagcagct gaatccttac     1980

ctgaggctga aggtgcggag agaccacatc atcgatgacg ccctggtgcg cctggagatg     2040

atcgccatgg agaatccagc cgatctgaag aagcagctgt acgtggagtt tgagggagag     2100

cagggagtgg acgagggagg cgtgtctaag gagttctttc agctggtggt ggaggagatc     2160

ttcaaccccg atatcggcat gtttacctac gacgagagca caaagctgtt ctggtttaat     2220

ccttcttcct tcgagaccga gggccagttt acactgatcg gcatcgtgct gggcctggcc     2280

atctacaaca attgtatcct ggacgtgcac ttcccaatgg tggtgtatag gaagctgatg     2340

ggcaagaagg gcacctttcg cgatctgggc gactcccacc ccgtgctgta ccagtctctg     2400

aaggatctgc tggagtatga gggcaacgtg gaggatgaca tgatgatcac cttccagatc     2460

tcccagacag acctgtttgg caacccaatg atgtacgatc tgaaggagaa cggcgacaag     2520

atccccatca caaacgagaa tagaaaggag ttcgtgaacc tgtacagcga ttatatcctg     2580

aataagtccg tggagaagca gttcaaggcc tttaggcgcg gcttccacat ggtgaccaac     2640

gagagccctc tgaagtatct gtttaggcca gaggagatcg agctgctgat ctgcggctcc     2700

cgcaatctgg actttcaggc cctggaggag accacagagt acgacggcgg ctataccagg     2760

gactctgtgc tgatccgcga gttctgggag atcgtgcaca gctttacaga cgagcagaag     2820

cggctgttcc tgcagtttac caccggcacc gacagagcac cagtgggagg actgggcaag     2880

ctgaagatga tcatcgccaa gaacggccca gacacagaga ggctgcccac cagccacacc     2940

tgtttcaacg tgctgctgct gcccgagtac tcctctaagg agaagctgaa ggagcgcctg     3000

ctgaaggcca tcacctacgc caagggcttt ggcatgctgt gatgaggtac cagtgaattc     3060

taccagtgcc ataggatagt gaattctacc agtgccatac acgtgagtga attctaccag     3120

tgccatagca tgcagtgaat tctaccagtg ccataggcgg ccgcttcgag cagacatgat     3180

aagatacatt gatgagtttg gacaaaccac aactagaatg cagtgaaaaa aatgctttat     3240

ttgtgaaatt tgtgatgcta ttgctttatt tgtaaccatt ataagctgca ataaacaagt     3300

taacaacaac aattgcattc attttatgtt tcaggttcag ggggagatgt gggaggtttt     3360

ttaaagcaag taaaacctct acaaatgtgg taaaatcg                             3398


<210>  23
<211>  3294
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  expression cassette  hSyn.hUbe3a-1.GSco.SV40


<220>
<221>  promoter
<222>  (1)..(466)
<223>  hSynapsin promoter

<220>
<221>  misc_feature
<222>  (478)..(483)
<223>  Kozak

<220>
<221>  misc_feature
<222>  (484)..(3045)
<223>  UBE3A-1

<220>
<221>  polyA_signal
<222>  (3063)..(3294)
<223>  SV40 late polyA

<400>  23
ctgcagaggg ccctgcgtat gagtgcaagt gggttttagg accaggatga ggcggggtgg       60

gggtgcctac ctgacgaccg accccgaccc actggacaag cacccaaccc ccattcccca      120

aattgcgcat cccctatcag agagggggag gggaaacagg atgcggcgag gcgcgtgcgc      180

actgccagct tcagcaccgc ggacagtgcc ttcgcccccg cctggcggcg cgcgccaccg      240

ccgcctcagc actgaaggcg cgctgacgtc actcgccggt cccccgcaaa ctccccttcc      300

cggccacctt ggtcgcgtcc gcgccgccgc cggcccagcc ggaccgcacc acgcgaggcg      360

cgagataggg gggcacgggc gcgaccatct gcgctgcggc gccggcgact cagcgctgcc      420

tcagtctgcg gtgggcagcg gaggagtcgt gtcgtgcctg agagcgcagt cgaattcgcc      480

accatgaaga gggcagcagc aaagcacctg atcgagagat actatcacca gctgaccgag      540

ggatgcggaa acgaggcatg tacaaacgag ttctgcgcct cctgtcccac ctttctgagg      600

atggataaca acgccgccgc catcaaggcc ctggagctgt acaagatcaa cgccaagctg      660

tgcgaccccc accctagcaa gaagggcgcc agctccgcct atctggagaa ctccaagggc      720

gcccctaaca atagctgttc cgagatcaag atgaataaga agggcgcccg gatcgatttc      780

aaggacgtga cctacctgac agaggagaag gtgtacgaga tcctggagct gtgccgggag      840

agagaggatt acagcccact gatcagagtg atcggcagag tgttctctag cgccgaggcc      900

ctggtgcagt cctttagaaa ggtgaagcag cacacaaagg aggagctgaa gtctctgcag      960

gccaaggacg aggacaagga cgaggacgag aaggagaagg cagcctgttc tgccgcagca     1020

atggaggagg acagcgaggc atcctctagc cggatcggcg attcctctca aggcgacaac     1080

aatctgcaga agctgggccc cgacgacgtg agcgtggata tcgacgccat ccggagagtg     1140

tacacaagac tgctgagcaa cgagaagatc gagaccgcct tcctgaacgc cctggtgtat     1200

ctgagcccta atgtggagtg cgatctgacc taccacaacg tgtactcccg ggacccaaac     1260

tacctgaatc tgttcatcat cgtgatggag aacagaaatc tgcactcccc cgagtatctg     1320

gagatggccc tgcctctgtt ttgtaaggcc atgtccaagc tgcctctggc agcacagggc     1380

aagctgatca ggctgtggtc taagtacaac gccgatcaga tcaggcgcat gatggagacc     1440

ttccagcagc tgatcacata caaagtgatc tctaacgagt ttaatagccg caacctggtg     1500

aacgacgacg acgccatcgt ggccgcctct aagtgcctga agatggtgta ctacgccaac     1560

gtggtgggcg gcgaggtgga cacaaaccac aacgaggagg acgacgagga gccaatcccc     1620

gagagctccg agctgaccct gcaggagctg ctgggagagg agcggagaaa taagaaggga     1680

ccaagggtgg atcctctgga gaccgagctg ggcgtgaaga cactggactg cagaaagcct     1740

ctgatcccat tcgaggagtt tatcaacgag cccctgaacg aggtgctgga gatggataag     1800

gactacacct tctttaaggt ggagacagag aacaagttca gctttatgac ctgtcctttc     1860

atcctgaacg ccgtgaccaa gaatctgggc ctgtactacg ataacaggat ccgcatgtac     1920

tccgagaggc gcatcaccgt gctgtattct ctggtgcagg gccagcagct gaatccttac     1980

ctgaggctga aggtgcggag agaccacatc atcgatgacg ccctggtgcg cctggagatg     2040

atcgccatgg agaatccagc cgatctgaag aagcagctgt acgtggagtt tgagggagag     2100

cagggagtgg acgagggagg cgtgtctaag gagttctttc agctggtggt ggaggagatc     2160

ttcaaccccg atatcggcat gtttacctac gacgagagca caaagctgtt ctggtttaat     2220

ccttcttcct tcgagaccga gggccagttt acactgatcg gcatcgtgct gggcctggcc     2280

atctacaaca attgtatcct ggacgtgcac ttcccaatgg tggtgtatag gaagctgatg     2340

ggcaagaagg gcacctttcg cgatctgggc gactcccacc ccgtgctgta ccagtctctg     2400

aaggatctgc tggagtatga gggcaacgtg gaggatgaca tgatgatcac cttccagatc     2460

tcccagacag acctgtttgg caacccaatg atgtacgatc tgaaggagaa cggcgacaag     2520

atccccatca caaacgagaa tagaaaggag ttcgtgaacc tgtacagcga ttatatcctg     2580

aataagtccg tggagaagca gttcaaggcc tttaggcgcg gcttccacat ggtgaccaac     2640

gagagccctc tgaagtatct gtttaggcca gaggagatcg agctgctgat ctgcggctcc     2700

cgcaatctgg actttcaggc cctggaggag accacagagt acgacggcgg ctataccagg     2760

gactctgtgc tgatccgcga gttctgggag atcgtgcaca gctttacaga cgagcagaag     2820

cggctgttcc tgcagtttac caccggcacc gacagagcac cagtgggagg actgggcaag     2880

ctgaagatga tcatcgccaa gaacggccca gacacagaga ggctgcccac cagccacacc     2940

tgtttcaacg tgctgctgct gcccgagtac tcctctaagg agaagctgaa ggagcgcctg     3000

ctgaaggcca tcacctacgc caagggcttt ggcatgctgt gatgaggtac cggcggccgc     3060

ttcgagcaga catgataaga tacattgatg agtttggaca aaccacaact agaatgcagt     3120

gaaaaaaatg ctttatttgt gaaatttgtg atgctattgc tttatttgta accattataa     3180

gctgcaataa acaagttaac aacaacaatt gcattcattt tatgtttcag gttcaggggg     3240

agatgtggga ggttttttaa agcaagtaaa acctctacaa atgtggtaaa atcg           3294


<210>  24
<211>  3467
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  expression cassette hSyn.hUbe3a-2.GSco.4XmiRNA183.SV40


<220>
<221>  promoter
<222>  (1)..(466)
<223>  hSynapsin

<220>
<221>  misc_feature
<222>  (478)..(483)
<223>  Kozak

<220>
<221>  misc_feature
<222>  (484)..(3114)
<223>  hUBE3A-2

<220>
<221>  misc_feature
<222>  (3121)..(3142)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3147)..(3168)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3175)..(3196)
<223>  miRNA183

<220>
<221>  misc_feature
<222>  (3203)..(3224)
<223>  miRNA183

<220>
<221>  polyA_signal
<222>  (3236)..(3467)
<223>  SV40 late polyA

<400>  24
ctgcagaggg ccctgcgtat gagtgcaagt gggttttagg accaggatga ggcggggtgg       60

gggtgcctac ctgacgaccg accccgaccc actggacaag cacccaaccc ccattcccca      120

aattgcgcat cccctatcag agagggggag gggaaacagg atgcggcgag gcgcgtgcgc      180

actgccagct tcagcaccgc ggacagtgcc ttcgcccccg cctggcggcg cgcgccaccg      240

ccgcctcagc actgaaggcg cgctgacgtc actcgccggt cccccgcaaa ctccccttcc      300

cggccacctt ggtcgcgtcc gcgccgccgc cggcccagcc ggaccgcacc acgcgaggcg      360

cgagataggg gggcacgggc gcgaccatct gcgctgcggc gccggcgact cagcgctgcc      420

tcagtctgcg gtgggcagcg gaggagtcgt gtcgtgcctg agagcgcagt cgaattcgcc      480

accatggaga agctgcacca gtgctactgg aagtctggcg agcctcagag cgacgatatc      540

gaggcaagca ggatgaagag agcagcagcc aagcacctga tcgagcggta ctatcaccag      600

ctgaccgagg gctgcggaaa cgaggcctgt acaaacgagt tctgcgcctc ctgtccaacc      660

tttctgagaa tggataacaa cgccgccgcc atcaaggccc tggagctgta caagatcaac      720

gccaagctgt gcgaccccca cccttctaag aagggcgcca gctccgccta tctggagaac      780

agcaagggcg cccccaacaa tagctgttcc gagatcaaga tgaataagaa gggcgccagg      840

atcgatttca aggacgtgac ctacctgaca gaggagaagg tgtacgagat cctggagctg      900

tgccgggaga gagaggatta ctcccctctg atcagagtga tcggacgcgt gttctctagc      960

gccgaggccc tggtgcagag ctttcgcaag gtgaagcagc acacaaagga ggagctgaag     1020

tccctgcagg ccaaggacga ggacaaggac gaggacgaga aggagaaggc agcttgttcc     1080

gccgcagcaa tggaggagga ctctgaggcc tcctctagca ggatcggcga ttcctctcaa     1140

ggcgacaaca atctgcagaa gctgggcccc gacgacgtga gcgtggatat cgacgccatc     1200

cggagagtgt acacacgcct gctgtctaac gagaagatcg agaccgcctt cctgaacgcc     1260

ctggtgtatc tgtctcctaa tgtggagtgc gatctgacct accacaacgt gtacagccgg     1320

gacccaaact acctgaatct gttcatcatc gtgatggaga acagaaatct gcactctccc     1380

gagtatctgg agatggccct gcctctgttt tgtaaggcca tgagcaagct gccactggca     1440

gcacagggca agctgatcag gctgtggtcc aagtacaacg ccgatcagat caggcgcatg     1500

atggagacct tccagcagct gatcacatat aaagtgatct ccaacgagtt taattctaga     1560

aacctggtga acgacgacga cgccatcgtg gccgccagca agtgcctgaa gatggtgtac     1620

tacgccaacg tggtgggcgg cgaggtggac acaaaccaca acgaggagga cgacgaggag     1680

ccaatccccg agagctccga gctgaccctg caggagctgc tgggagagga gcggagaaat     1740

aagaagggac caagggtgga tcctctggag accgagctgg gcgtgaagac actggactgc     1800

agaaagcctc tgatcccatt cgaggagttt atcaacgagc ccctgaacga ggtgctggag     1860

atggataagg actacacctt ctttaaggtg gagacagaga acaagttcag ctttatgacc     1920

tgtcctttca tcctgaacgc cgtgaccaag aatctgggcc tgtactacga taacaggatc     1980

cgcatgtaca gcgagaggcg catcaccgtg ctgtattccc tggtgcaggg ccagcagctg     2040

aatccttacc tgaggctgaa ggtgcggaga gaccacatca tcgatgacgc cctggtgcgc     2100

ctggagatga tcgccatgga gaatccagcc gatctgaaga agcagctgta cgtggagttt     2160

gagggagagc agggagtgga cgagggaggc gtgtccaagg agttctttca gctggtggtg     2220

gaggagatct tcaaccccga tatcggcatg tttacctacg acgagtctac aaagctgttc     2280

tggtttaatc cttcttcctt cgagaccgag ggccagttta cactgatcgg catcgtgctg     2340

ggcctggcca tctacaacaa ttgtatcctg gacgtgcact tcccaatggt ggtgtatagg     2400

aagctgatgg gcaagaaggg cacctttcgc gatctgggcg acagccaccc cgtgctgtac     2460

cagtccctga aggatctgct ggagtatgag ggcaacgtgg aggatgacat gatgatcacc     2520

ttccagatct cccagacaga cctgtttggc aacccaatga tgtacgatct gaaggagaac     2580

ggcgacaaga tccccatcac aaacgagaat aggaaggagt tcgtgaacct gtactctgat     2640

tatatcctga ataagagcgt ggagaagcag ttcaaggcct ttaggcgcgg cttccacatg     2700

gtgaccaacg agtctcctct gaagtatctg tttaggccag aggagatcga gctgctgatc     2760

tgcggcagcc gcaatctgga ctttcaggcc ctggaggaga ccacagagta cgacggcggc     2820

tatacccggg actccgtgct gatcagagag ttctgggaga tcgtgcactc ttttacagac     2880

gagcagaagc ggctgttcct gcagtttacc accggcaccg acagagcacc agtgggagga     2940

ctgggcaagc tgaagatgat catcgccaag aacggcccag acacagagag gctgcccacc     3000

agccacacct gtttcaacgt gctgctgctg cccgagtact cctctaagga gaagctgaag     3060

gagcgcctgc tgaaggccat cacctacgcc aagggctttg gcatgctgtg atgaggtacc     3120

agtgaattct accagtgcca taggatagtg aattctacca gtgccataca cgtgagtgaa     3180

ttctaccagt gccatagcat gcagtgaatt ctaccagtgc cataggcggc cgcttcgagc     3240

agacatgata agatacattg atgagtttgg acaaaccaca actagaatgc agtgaaaaaa     3300

atgctttatt tgtgaaattt gtgatgctat tgctttattt gtaaccatta taagctgcaa     3360

taaacaagtt aacaacaaca attgcattca ttttatgttt caggttcagg gggagatgtg     3420

ggaggttttt taaagcaagt aaaacctcta caaatgtggt aaaatcg                   3467


<210>  25
<211>  3363
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  expression cassette hSyn.hUbe3a-2.GSco.SV40


<220>
<221>  promoter
<222>  (1)..(466)
<223>  hSynapsin

<220>
<221>  misc_feature
<222>  (478)..(483)
<223>  Kozak

<220>
<221>  misc_feature
<222>  (484)..(3114)
<223>  hUBE3A-2

<220>
<221>  polyA_signal
<222>  (3132)..(3363)
<223>  SV40 PolyA

<400>  25
ctgcagaggg ccctgcgtat gagtgcaagt gggttttagg accaggatga ggcggggtgg       60

gggtgcctac ctgacgaccg accccgaccc actggacaag cacccaaccc ccattcccca      120

aattgcgcat cccctatcag agagggggag gggaaacagg atgcggcgag gcgcgtgcgc      180

actgccagct tcagcaccgc ggacagtgcc ttcgcccccg cctggcggcg cgcgccaccg      240

ccgcctcagc actgaaggcg cgctgacgtc actcgccggt cccccgcaaa ctccccttcc      300

cggccacctt ggtcgcgtcc gcgccgccgc cggcccagcc ggaccgcacc acgcgaggcg      360

cgagataggg gggcacgggc gcgaccatct gcgctgcggc gccggcgact cagcgctgcc      420

tcagtctgcg gtgggcagcg gaggagtcgt gtcgtgcctg agagcgcagt cgaattcgcc      480

accatggaga agctgcacca gtgctactgg aagtctggcg agcctcagag cgacgatatc      540

gaggcaagca ggatgaagag agcagcagcc aagcacctga tcgagcggta ctatcaccag      600

ctgaccgagg gctgcggaaa cgaggcctgt acaaacgagt tctgcgcctc ctgtccaacc      660

tttctgagaa tggataacaa cgccgccgcc atcaaggccc tggagctgta caagatcaac      720

gccaagctgt gcgaccccca cccttctaag aagggcgcca gctccgccta tctggagaac      780

agcaagggcg cccccaacaa tagctgttcc gagatcaaga tgaataagaa gggcgccagg      840

atcgatttca aggacgtgac ctacctgaca gaggagaagg tgtacgagat cctggagctg      900

tgccgggaga gagaggatta ctcccctctg atcagagtga tcggacgcgt gttctctagc      960

gccgaggccc tggtgcagag ctttcgcaag gtgaagcagc acacaaagga ggagctgaag     1020

tccctgcagg ccaaggacga ggacaaggac gaggacgaga aggagaaggc agcttgttcc     1080

gccgcagcaa tggaggagga ctctgaggcc tcctctagca ggatcggcga ttcctctcaa     1140

ggcgacaaca atctgcagaa gctgggcccc gacgacgtga gcgtggatat cgacgccatc     1200

cggagagtgt acacacgcct gctgtctaac gagaagatcg agaccgcctt cctgaacgcc     1260

ctggtgtatc tgtctcctaa tgtggagtgc gatctgacct accacaacgt gtacagccgg     1320

gacccaaact acctgaatct gttcatcatc gtgatggaga acagaaatct gcactctccc     1380

gagtatctgg agatggccct gcctctgttt tgtaaggcca tgagcaagct gccactggca     1440

gcacagggca agctgatcag gctgtggtcc aagtacaacg ccgatcagat caggcgcatg     1500

atggagacct tccagcagct gatcacatat aaagtgatct ccaacgagtt taattctaga     1560

aacctggtga acgacgacga cgccatcgtg gccgccagca agtgcctgaa gatggtgtac     1620

tacgccaacg tggtgggcgg cgaggtggac acaaaccaca acgaggagga cgacgaggag     1680

ccaatccccg agagctccga gctgaccctg caggagctgc tgggagagga gcggagaaat     1740

aagaagggac caagggtgga tcctctggag accgagctgg gcgtgaagac actggactgc     1800

agaaagcctc tgatcccatt cgaggagttt atcaacgagc ccctgaacga ggtgctggag     1860

atggataagg actacacctt ctttaaggtg gagacagaga acaagttcag ctttatgacc     1920

tgtcctttca tcctgaacgc cgtgaccaag aatctgggcc tgtactacga taacaggatc     1980

cgcatgtaca gcgagaggcg catcaccgtg ctgtattccc tggtgcaggg ccagcagctg     2040

aatccttacc tgaggctgaa ggtgcggaga gaccacatca tcgatgacgc cctggtgcgc     2100

ctggagatga tcgccatgga gaatccagcc gatctgaaga agcagctgta cgtggagttt     2160

gagggagagc agggagtgga cgagggaggc gtgtccaagg agttctttca gctggtggtg     2220

gaggagatct tcaaccccga tatcggcatg tttacctacg acgagtctac aaagctgttc     2280

tggtttaatc cttcttcctt cgagaccgag ggccagttta cactgatcgg catcgtgctg     2340

ggcctggcca tctacaacaa ttgtatcctg gacgtgcact tcccaatggt ggtgtatagg     2400

aagctgatgg gcaagaaggg cacctttcgc gatctgggcg acagccaccc cgtgctgtac     2460

cagtccctga aggatctgct ggagtatgag ggcaacgtgg aggatgacat gatgatcacc     2520

ttccagatct cccagacaga cctgtttggc aacccaatga tgtacgatct gaaggagaac     2580

ggcgacaaga tccccatcac aaacgagaat aggaaggagt tcgtgaacct gtactctgat     2640

tatatcctga ataagagcgt ggagaagcag ttcaaggcct ttaggcgcgg cttccacatg     2700

gtgaccaacg agtctcctct gaagtatctg tttaggccag aggagatcga gctgctgatc     2760

tgcggcagcc gcaatctgga ctttcaggcc ctggaggaga ccacagagta cgacggcggc     2820

tatacccggg actccgtgct gatcagagag ttctgggaga tcgtgcactc ttttacagac     2880

gagcagaagc ggctgttcct gcagtttacc accggcaccg acagagcacc agtgggagga     2940

ctgggcaagc tgaagatgat catcgccaag aacggcccag acacagagag gctgcccacc     3000

agccacacct gtttcaacgt gctgctgctg cccgagtact cctctaagga gaagctgaag     3060

gagcgcctgc tgaaggccat cacctacgcc aagggctttg gcatgctgtg atgaggtacc     3120

ggcggccgct tcgagcagac atgataagat acattgatga gtttggacaa accacaacta     3180

gaatgcagtg aaaaaaatgc tttatttgtg aaatttgtga tgctattgct ttatttgtaa     3240

ccattataag ctgcaataaa caagttaaca acaacaattg cattcatttt atgtttcagg     3300

ttcaggggga gatgtgggag gttttttaaa gcaagtaaaa cctctacaaa tgtggtaaaa     3360

tcg                                                                   3363


