                         SEQUENCE LISTING

<110>  Research Institute at Nationwide Children's Hospital
 
<120>  ADENO-ASSOCIATED VIRUS DELIVERY OF CLN3 POLYNUCLEOTIDE

<130>  33057/53576 PC

<150>  US 62/800,911
<151>  2019-02-04

<160>  12    

<170>  PatentIn version 3.5

<210>  1
<211>  438
<212>  PRT
<213>  Homo sapiens


<220>
<221>  MISC_FEATURE
<223>  CLN3

<400>  1

Met Gly Gly Cys Ala Gly Ser Arg Arg Arg Phe Ser Asp Ser Glu Gly 
1               5                   10                  15      


Glu Glu Thr Val Pro Glu Pro Arg Leu Pro Leu Leu Asp His Gln Gly 
            20                  25                  30          


Ala His Trp Lys Asn Ala Val Gly Phe Trp Leu Leu Gly Leu Cys Asn 
        35                  40                  45              


Asn Phe Ser Tyr Val Val Met Leu Ser Ala Ala His Asp Ile Leu Ser 
    50                  55                  60                  


His Lys Arg Thr Ser Gly Asn Gln Ser His Val Asp Pro Gly Pro Thr 
65                  70                  75                  80  


Pro Ile Pro His Asn Ser Ser Ser Arg Phe Asp Cys Asn Ser Val Ser 
                85                  90                  95      


Thr Ala Ala Val Leu Leu Ala Asp Ile Leu Pro Thr Leu Val Ile Lys 
            100                 105                 110         


Leu Leu Ala Pro Leu Gly Leu His Leu Leu Pro Tyr Ser Pro Arg Val 
        115                 120                 125             


Leu Val Ser Gly Ile Cys Ala Ala Gly Ser Phe Val Leu Val Ala Phe 
    130                 135                 140                 


Ser His Ser Val Gly Thr Ser Leu Cys Gly Val Val Phe Ala Ser Ile 
145                 150                 155                 160 


Ser Ser Gly Leu Gly Glu Val Thr Phe Leu Ser Leu Thr Ala Phe Tyr 
                165                 170                 175     


Pro Arg Ala Val Ile Ser Trp Trp Ser Ser Gly Thr Gly Gly Ala Gly 
            180                 185                 190         


Leu Leu Gly Ala Leu Ser Tyr Leu Gly Leu Thr Gln Ala Gly Leu Ser 
        195                 200                 205             


Pro Gln Gln Thr Leu Leu Ser Met Leu Gly Ile Pro Ala Leu Leu Leu 
    210                 215                 220                 


Ala Ser Tyr Phe Leu Leu Leu Thr Ser Pro Glu Ala Gln Asp Pro Gly 
225                 230                 235                 240 


Gly Glu Glu Glu Ala Glu Ser Ala Ala Arg Gln Pro Leu Ile Arg Thr 
                245                 250                 255     


Glu Ala Pro Glu Ser Lys Pro Gly Ser Ser Ser Ser Leu Ser Leu Arg 
            260                 265                 270         


Glu Arg Trp Thr Val Phe Lys Gly Leu Leu Trp Tyr Ile Val Pro Leu 
        275                 280                 285             


Val Val Val Tyr Phe Ala Glu Tyr Phe Ile Asn Gln Gly Leu Phe Glu 
    290                 295                 300                 


Leu Leu Phe Phe Trp Asn Thr Ser Leu Ser His Ala Gln Gln Tyr Arg 
305                 310                 315                 320 


Trp Tyr Gln Met Leu Tyr Gln Ala Gly Val Phe Ala Ser Arg Ser Ser 
                325                 330                 335     


Leu Arg Cys Cys Arg Ile Arg Phe Thr Trp Ala Leu Ala Leu Leu Gln 
            340                 345                 350         


Cys Leu Asn Leu Val Phe Leu Leu Ala Asp Val Trp Phe Gly Phe Leu 
        355                 360                 365             


Pro Ser Ile Tyr Leu Val Phe Leu Ile Ile Leu Tyr Glu Gly Leu Leu 
    370                 375                 380                 


Gly Gly Ala Ala Tyr Val Asn Thr Phe His Asn Ile Ala Leu Glu Thr 
385                 390                 395                 400 


Ser Asp Glu His Arg Glu Phe Ala Met Ala Ala Thr Cys Ile Ser Asp 
                405                 410                 415     


Thr Leu Gly Ile Ser Leu Ser Gly Leu Leu Ala Leu Pro Leu His Asp 
            420                 425                 430         


Phe Leu Cys Gln Leu Ser 
        435             


<210>  2
<211>  1317
<212>  DNA
<213>  Homo sapiens


<220>
<221>  misc_feature
<223>  CNL3

<400>  2
atgggaggct gtgcaggctc gcggcggcgc ttttcggatt ccgaggggga ggagaccgtc       60

ccggagcccc ggctccctct gttggaccat cagggcgcgc attggaagaa cgcggtgggc      120

ttctggctgc tgggcctttg caacaacttc tcttatgtgg tgatgctgag tgccgcccac      180

gacatcctta gccacaagag gacatcggga aaccagagcc atgtggaccc aggcccaacg      240

ccgatccccc acaacagctc atcacgattt gactgcaact ctgtctctac ggctgctgtg      300

ctcctggcgg acatcctccc cacactcgtc atcaaattgt tggctcctct tggccttcac      360

ctgctgccct acagcccccg ggttctcgtc agtgggattt gtgctgctgg aagcttcgtc      420

ctggttgcct tttctcattc tgtggggacc agcctgtgtg gtgtggtctt cgctagcatc      480

tcatcaggcc ttggggaggt caccttcctc tccctcactg ccttctaccc cagggccgtg      540

atctcctggt ggtcctcagg gactggggga gctgggctgc tgggggccct gtcctacctg      600

ggcctcaccc aggccggcct ctcccctcag cagaccctgc tgtccatgct gggtatccct      660

gccctgctgc tggccagcta tttcttgttg ctcacatctc ctgaggccca ggaccctgga      720

ggggaagaag aagcagagag cgcagcccgg cagcccctca taagaaccga ggccccggag      780

tcgaagccag gctccagctc cagcctctcc cttcgggaaa ggtggacagt gttcaagggt      840

ctgctgtggt acattgttcc cttggtcgta gtttactttg ccgagtattt cattaaccag      900

ggactttttg aactcctctt tttctggaac acttccctga gtcacgctca gcaataccgc      960

tggtaccaga tgctgtacca ggctggcgtc tttgcctccc gctcttctct ccgctgctgt     1020

cgcatccgtt tcacctgggc cctggccctg ctgcagtgcc tcaacctggt gttcctgctg     1080

gcagacgtgt ggttcggctt tctgccaagc atctacctcg tcttcctgat cattctgtat     1140

gaggggctcc tgggaggcgc agcctacgtg aacaccttcc acaacatcgc cctggagacc     1200

agtgatgagc accgggagtt tgcaatggcg gccacctgca tctctgacac actggggatc     1260

tccctgtcgg ggctcctggc tttgcctctg catgacttcc tctgccagct ctcctga        1317


<210>  3
<211>  546
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic Polynucleotide


<220>
<221>  misc_feature
<223>  P546 promoter

<400>  3
gaacaacgcc aggctcctca acaggcaact ttgctacttc tacagaaaat gataataaag       60

aaatgctggt gaagtcaaat gcttatcaca atggtgaact actcagcagg gaggctctaa      120

taggcgccaa gagcctagac ttccttaagc gccagagtcc acaagggccc agttaatcct      180

caacattcaa atgctgccca caaaaccagc ccctctgtgc cctagccgcc tcttttttcc      240

aagtgacagt agaactccac caatccgcag ctgaatgggg tccgcctctt ttccctgcct      300

aaacagacag gaactcctgc caattgaggg cgtcaccgct aaggctccgc cccagcctgg      360

gctccacaac caatgaaggg taatctcgac aaagagcaag gggtggggcg cgggcgcgca      420

ggtgcagcag cacacaggct ggtcgggagg gcggggcgcg acgtctgccg tgcggggtcc      480

cggcatcggt tgcgcgcgcg ctccctcctc tcggagagag ggctgtggta aaacccgtcc      540

ggaaaa                                                                 546


<210>  4
<211>  2479
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic Polynucleotide


<220>
<221>  misc_feature
<223>  CLN3 gene cassette

<400>  4
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggaatt caattcacgc      120

gccggtaccg aattcacgcg tgaacaacgc caggctcctc aacaggcaac tttgctactt      180

ctacagaaaa tgataataaa gaaatgctgg tgaagtcaaa tgcttatcac aatggtgaac      240

tactcagcag ggaggctcta ataggcgcca agagcctaga cttccttaag cgccagagtc      300

cacaagggcc cagttaatcc tcaacattca aatgctgccc acaaaaccag cccctctgtg      360

ccctagccgc ctcttttttc caagtgacag tagaactcca ccaatccgca gctgaatggg      420

gtccgcctct tttccctgcc taaacagaca ggaactcctg ccaattgagg gcgtcaccgc      480

taaggctccg ccccagcctg ggctccacaa ccaatgaagg gtaatctcga caaagagcaa      540

ggggtggggc gcgggcgcgc aggtgcagca gcacacaggc tggtcgggag ggcggggcgc      600

gacgtctgcc gtgcggggtc ccggcatcgg ttgcgcgcgc gctccctcct ctcggagaga      660

gggctgtggt aaaacccgtc cggaaaacgc gtcgaagggc gaattctgca gataactggt      720

aagtttagtc ttttttgtct tttatttcag gtcccggatc cggtggtggt gcaaatcaaa      780

gaactgctcc tcagtcgatg ttgcctttac ttctaggcct gtacggaagt gttactaccg      840

gtatgggagg ctgtgcaggc tcgcggcggc gcttttcgga ttccgagggg gaggagaccg      900

tcccggagcc ccggctccct ctgttggacc atcagggcgc gcattggaag aacgcggtgg      960

gcttctggct gctgggcctt tgcaacaact tctcttatgt ggtgatgctg agtgccgccc     1020

acgacatcct tagccacaag aggacatcgg gaaaccagag ccatgtggac ccaggcccaa     1080

cgccgatccc ccacaacagc tcatcacgat ttgactgcaa ctctgtctct acggctgctg     1140

tgctcctggc ggacatcctc cccacactcg tcatcaaatt gttggctcct cttggccttc     1200

acctgctgcc ctacagcccc cgggttctcg tcagtgggat ttgtgctgct ggaagcttcg     1260

tcctggttgc cttttctcat tctgtgggga ccagcctgtg tggtgtggtc ttcgctagca     1320

tctcatcagg ccttggggag gtcaccttcc tctccctcac tgccttctac cccagggccg     1380

tgatctcctg gtggtcctca gggactgggg gagctgggct gctgggggcc ctgtcctacc     1440

tgggcctcac ccaggccggc ctctcccctc agcagaccct gctgtccatg ctgggtatcc     1500

ctgccctgct gctggccagc tatttcttgt tgctcacatc tcctgaggcc caggaccctg     1560

gaggggaaga agaagcagag agcgcagccc ggcagcccct cataagaacc gaggccccgg     1620

agtcgaagcc aggctccagc tccagcctct cccttcggga aaggtggaca gtgttcaagg     1680

gtctgctgtg gtacattgtt cccttggtcg tagtttactt tgccgagtat ttcattaacc     1740

agggactttt tgaactcctc tttttctgga acacttccct gagtcacgct cagcaatacc     1800

gctggtacca gatgctgtac caggctggcg tctttgcctc ccgctcttct ctccgctgct     1860

gtcgcatccg tttcacctgg gccctggccc tgctgcagtg cctcaacctg gtgttcctgc     1920

tggcagacgt gtggttcggc tttctgccaa gcatctacct cgtcttcctg atcattctgt     1980

atgaggggct cctgggaggc gcagcctacg tgaacacctt ccacaacatc gccctggaga     2040

ccagtgatga gcaccgggag tttgcaatgg cggccacctg catctctgac acactgggga     2100

tctccctgtc ggggctcctg gctttgcctc tgcatgactt cctctgccag ctctcctgac     2160

ctgcaggcct cgactgtgcc ttctagttgc cagccatctg ttgtttgccc ctcccccgtg     2220

ccttccttga ccctggaagg tgccactccc actgtccttt cctaataaaa tgaggaaatt     2280

gcatcgcatt gtctgagtag gtgtcattct attcgcatgc tggggagaga tcgatctgag     2340

gaacccctag tgatggagtt ggccactccc tctctgcgcg ctcgctcgct cactgaggcc     2400

gggcgaccaa aggtcgcccg acgcccgggc tttgcccggg cggcctcagt gagcgagcga     2460

gcgcgcagag agggagtgg                                                  2479


<210>  5
<211>  6008
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic Polynucleotide


<220>
<221>  misc_feature
<223>  pAAV.P546.CLN3.KAN plasmid

<400>  5
gcccaatacg caaaccgcct ctccccgcgc gttggccgat tcattaatgc agctggcgta       60

atagcgaaga ggcccgcacc gatcgccctt cccaacagtt gcgcagcctg aatggcgaat      120

ggcgattccg ttgcaatggc tggcggtaat attgttctgg atattaccag caaggccgat      180

agtttgagtt cttctactca ggcaagtgat gttattacta atcaaagaag tattgcgaca      240

acggttaatt tgcgtgatgg acagactctt ttactcggtg gcctcactga ttataaaaac      300

acttctcagg attctggcgt accgttcctg tctaaaatcc ctttaatcgg cctcctgttt      360

agctcccgct ctgattctaa cgaggaaagc acgttatacg tgctcgtcaa agcaaccata      420

gtacgcgccc tgtagcggcg cattaagcgc ggcgggtgtg gtggttacgc gcagcgtgac      480

cgctacactt gccagcgccc tagcgcccgc tcctttcgct ttcttccctt cctttctcgc      540

cacgttcgcc ggctttcccc gtcaagctct aaatcggggg ctccctttag ggttccgatt      600

tagtgcttta cggcacctcg accccaaaaa acttgattag ggtgatggtt cacgtagtgg      660

gccatcgccc tgatagacgg tttttcgccc tttgacgttg gagtccacgt tctttaatag      720

tggactcttg ttccaaactg gaacaacact caaccctatc tcggtctatt cttttgattt      780

ataagggatt ttgccgattt cggcctattg gttaaaaaat gagctgattt aacaaaaatt      840

taacgcgaat tttaacaaaa tattaacgct tacaatttaa atatttgctt atacaatctt      900

cctgtttttg gggcttttct gattatcaac cggggtacat atgattgaca tgctagtttt      960

acgattaccg ttcatcgccc tgcgcgctcg ctcgctcact gaggccgccc gggcaaagcc     1020

cgggcgtcgg gcgacctttg gtcgcccggc ctcagtgagc gagcgagcgc gcagagaggg     1080

agtggaattc aattcacgcg ccggtaccga attcacgcgt gaacaacgcc aggctcctca     1140

acaggcaact ttgctacttc tacagaaaat gataataaag aaatgctggt gaagtcaaat     1200

gcttatcaca atggtgaact actcagcagg gaggctctaa taggcgccaa gagcctagac     1260

ttccttaagc gccagagtcc acaagggccc agttaatcct caacattcaa atgctgccca     1320

caaaaccagc ccctctgtgc cctagccgcc tcttttttcc aagtgacagt agaactccac     1380

caatccgcag ctgaatgggg tccgcctctt ttccctgcct aaacagacag gaactcctgc     1440

caattgaggg cgtcaccgct aaggctccgc cccagcctgg gctccacaac caatgaaggg     1500

taatctcgac aaagagcaag gggtggggcg cgggcgcgca ggtgcagcag cacacaggct     1560

ggtcgggagg gcggggcgcg acgtctgccg tgcggggtcc cggcatcggt tgcgcgcgcg     1620

ctccctcctc tcggagagag ggctgtggta aaacccgtcc ggaaaacgcg tcgaagggcg     1680

aattctgcag ataactggta agtttagtct tttttgtctt ttatttcagg tcccggatcc     1740

ggtggtggtg caaatcaaag aactgctcct cagtcgatgt tgcctttact tctaggcctg     1800

tacggaagtg ttactaccgg tatgggaggc tgtgcaggct cgcggcggcg cttttcggat     1860

tccgaggggg aggagaccgt cccggagccc cggctccctc tgttggacca tcagggcgcg     1920

cattggaaga acgcggtggg cttctggctg ctgggccttt gcaacaactt ctcttatgtg     1980

gtgatgctga gtgccgccca cgacatcctt agccacaaga ggacatcggg aaaccagagc     2040

catgtggacc caggcccaac gccgatcccc cacaacagct catcacgatt tgactgcaac     2100

tctgtctcta cggctgctgt gctcctggcg gacatcctcc ccacactcgt catcaaattg     2160

ttggctcctc ttggccttca cctgctgccc tacagccccc gggttctcgt cagtgggatt     2220

tgtgctgctg gaagcttcgt cctggttgcc ttttctcatt ctgtggggac cagcctgtgt     2280

ggtgtggtct tcgctagcat ctcatcaggc cttggggagg tcaccttcct ctccctcact     2340

gccttctacc ccagggccgt gatctcctgg tggtcctcag ggactggggg agctgggctg     2400

ctgggggccc tgtcctacct gggcctcacc caggccggcc tctcccctca gcagaccctg     2460

ctgtccatgc tgggtatccc tgccctgctg ctggccagct atttcttgtt gctcacatct     2520

cctgaggccc aggaccctgg aggggaagaa gaagcagaga gcgcagcccg gcagcccctc     2580

ataagaaccg aggccccgga gtcgaagcca ggctccagct ccagcctctc ccttcgggaa     2640

aggtggacag tgttcaaggg tctgctgtgg tacattgttc ccttggtcgt agtttacttt     2700

gccgagtatt tcattaacca gggacttttt gaactcctct ttttctggaa cacttccctg     2760

agtcacgctc agcaataccg ctggtaccag atgctgtacc aggctggcgt ctttgcctcc     2820

cgctcttctc tccgctgctg tcgcatccgt ttcacctggg ccctggccct gctgcagtgc     2880

ctcaacctgg tgttcctgct ggcagacgtg tggttcggct ttctgccaag catctacctc     2940

gtcttcctga tcattctgta tgaggggctc ctgggaggcg cagcctacgt gaacaccttc     3000

cacaacatcg ccctggagac cagtgatgag caccgggagt ttgcaatggc ggccacctgc     3060

atctctgaca cactggggat ctccctgtcg gggctcctgg ctttgcctct gcatgacttc     3120

ctctgccagc tctcctgacc tgcaggcctc gactgtgcct tctagttgcc agccatctgt     3180

tgtttgcccc tcccccgtgc cttccttgac cctggaaggt gccactccca ctgtcctttc     3240

ctaataaaat gaggaaattg catcgcattg tctgagtagg tgtcattcta ttcgcatgct     3300

ggggagagat cgatctgagg aacccctagt gatggagttg gccactccct ctctgcgcgc     3360

tcgctcgctc actgaggccg ggcgaccaaa ggtcgcccga cgcccgggct ttgcccgggc     3420

ggcctcagtg agcgagcgag cgcgcagaga gggagtggcc cccccccccc cccccccggc     3480

gattctcttg tttgctccag actctcaggc aatgacctga tagcctttgt agagacctct     3540

caaaaatagc taccctctcc ggcatgaatt tatcagctag aacggttgaa tatcatattg     3600

atggtgattt gactgtctcc ggcctttctc acccgtttga atctttacct acacattact     3660

caggcattgc atttaaaata tatgagggtt ctaaaaattt ttatccttgc gttgaaataa     3720

aggcttctcc cgcaaaagta ttacagggtc ataatgtttt tggtacaacc gatttagctt     3780

tatgctctga ggctttattg cttaattttg ctaattcttt gccttgcctg tatgatttat     3840

tggatgttgg aatcgcctga tgcggtattt tctccttacg catctgtgcg gtatttcaca     3900

ccgcatatgg tgcactctca gtacaatctg ctctgatgcc gcatagttaa gccagccccg     3960

acacccgcca acactatggt gcactctcag tacaatctgc tctgatgccg catagttaag     4020

ccagccccga cacccgccaa cacccgctga cgcgccctga cgggcttgtc tgctcccggc     4080

atccgcttac agacaagctg tgaccgtctc cgggagctgc atgtgtcaga ggttttcacc     4140

gtcatcaccg aaacgcgcga gacgaaaggg cctcgtgata cgcctatttt tataggttaa     4200

tgtcatgaga caataaccct gataaatgct tcaataatat tgaaaaagga agagtatgag     4260

ccatattcaa cgggaaacgt cgaggccgcg attaaattcc aacatggatg ctgatttata     4320

tgggtataaa tgggctcgcg ataatgtcgg gcaatcaggt gcgacaatct atcgcttgta     4380

tgggaagccc gatgcgccag agttgtttct gaaacatggc aaaggtagcg ttgccaatga     4440

tgttacagat gagatggtca gactaaactg gctgacggaa tttatgccac ttccgaccat     4500

caagcatttt atccgtactc ctgatgatgc atggttactc accactgcga tccccggaaa     4560

aacagcgttc caggtattag aagaatatcc tgattcaggt gaaaatattg ttgatgcgct     4620

ggcagtgttc ctgcgccggt tgcactcgat tcctgtttgt aattgtcctt ttaacagcga     4680

tcgcgtattt cgcctcgctc aggcgcaatc acgaatgaat aacggtttgg ttgatgcgag     4740

tgattttgat gacgagcgta atggctggcc tgttgaacaa gtctggaaag aaatgcataa     4800

acttttgcca ttctcaccgg attcagtcgt cactcatggt gatttctcac ttgataacct     4860

tatttttgac gaggggaaat taataggttg tattgatgtt ggacgagtcg gaatcgcaga     4920

ccgataccag gatcttgcca tcctatggaa ctgcctcggt gagttttctc cttcattaca     4980

gaaacggctt tttcaaaaat atggtattga taatcctgat atgaataaat tgcagtttca     5040

tttgatgctc gatgagtttt tctaactgtc agaccaagtt tactcatata tactttagat     5100

tgatttaaaa cttcattttt aatttaaaag gatctaggtg aagatccttt ttgataatct     5160

catgaccaaa atcccttaac gtgagttttc gttccactga gcgtcagacc ccgtagaaaa     5220

gatcaaagga tcttcttgag atcctttttt tctgcgcgta atctgctgct tgcaaacaaa     5280

aaaaccaccg ctaccagcgg tggtttgttt gccggatcaa gagctaccaa ctctttttcc     5340

gaaggtaact ggcttcagca gagcgcagat accaaatact gttcttctag tgtagccgta     5400

gttaggccac cacttcaaga actctgtagc accgcctaca tacctcgctc tgctaatcct     5460

gttaccagtg gctgctgcca gtggcgataa gtcgtgtctt accgggttgg actcaagacg     5520

atagttaccg gataaggcgc agcggtcggg ctgaacgggg ggttcgtgca cacagcccag     5580

cttggagcga acgacctaca ccgaactgag atacctacag cgtgagctat gagaaagcgc     5640

cacgcttccc gaagggagaa aggcggacag gtatccggta agcggcaggg tcggaacagg     5700

agagcgcacg agggagcttc cagggggaaa cgcctggtat ctttatagtc ctgtcgggtt     5760

tcgccacctc tgacttgagc gtcgattttt gtgatgctcg tcaggggggc ggagcctatg     5820

gaaaaacgcc agcaacgcgg cctttttacg gttcctggcc ttttgctggc cttttgctca     5880

catgttcttt cctgcgttat cccctgattc tgtggataac cgtattaccg cctttgagtg     5940

agctgatacc gctcgccgca gccgaacgac cgagcgcagc gagtcagtga gcgaggaagc     6000

ggaagagc                                                              6008


<210>  6
<211>  106
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic Polynucleotide


<220>
<221>  misc_feature
<223>  5' ITR

<400>  6
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtgg                     106


<210>  7
<211>  100
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic Polynucleotide


<220>
<221>  misc_feature
<223>  SV40 Intron

<400>  7
ggtaagttta gtcttttttg tcttttattt caggtcccgg atccggtggt ggtgcaaatc       60

aaagaactgc tcctcagtcg atgttgcctt tacttctagg                            100


<210>  8
<211>  147
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic Polynucleotide


<220>
<221>  misc_feature
<223>  BGH Poly A

<400>  8
cctcgactgt gccttctagt tgccagccat ctgttgtttg cccctccccc gtgccttcct       60

tgaccctgga aggtgccact cccactgtcc tttcctaata aaatgaggaa attgcatcgc      120

attgtctgag taggtgtcat tctattc                                          147


<210>  9
<211>  141
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic Polynucleotide


<220>
<221>  misc_feature
<223>  3' ITR

<400>  9
aggaacccct agtgatggag ttggccactc cctctctgcg cgctcgctcg ctcactgagg       60

ccgggcgacc aaaggtcgcc cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc      120

gagcgcgcag agagggagtg g                                                141


<210>  10
<211>  736
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  Synthetic Polypeptide


<220>
<221>  MISC_FEATURE
<223>  scAAV9 seuqence

<400>  10

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 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 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 Ala 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>  11
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic Polynucleotide

<400>  11
cgctagcatc tcatcaggcc ttg                                               23


<210>  12
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Synthetic Polynucleotide

<400>  12
agcatggaca gcagggtctg                                                   20


