                         SEQUENCE LISTING

<110>  FUNDACION PARA LA INVESTIGACION MEDICA APLICADA
 
<120>  VIRAL PARTICLES FOR USE IN TREATING SYNUCLEINOPATHIES SUCH AS 
       PARKINSON'S DISEASES BY GENE THERAPY

<130>  BCT200202QT

<150>  EP19382706
<151>  2019-08-12

<160>  28    

<170>  PatentIn version 3.5

<210>  1
<211>  1551
<212>  DNA
<213>  Homo sapiens

<400>  1
atggctggca gtcttacagg tctcctgctc ctgcaagctg tctcttgggc ttctggggcc       60

aggccctgta tccccaaatc ctttggatac tcatctgtgg tgtgtgtttg taatgccact      120

tattgtgata gctttgaccc ccccaccttt cctgcactgg gcaccttttc aaggtatgaa      180

tctaccaggt ctgggaggag gatggagctg agtatggggc ccatccaagc aaaccatact      240

ggcactggct tgctgctgac actgcaacct gaacagaagt tccagaaagt gaagggcttt      300

ggaggagcca tgactgatgc tgctgccctc aatattttgg ccctgagccc ccctgctcag      360

aatctccttt tgaaatcata cttctctgag gagggaattg gatacaatat catcagggtg      420

ccaatggcct catgtgactt tagtattagg acttacacct atgctgatac ccctgatgat      480

ttccagctgc ataacttctc attgcctgag gaggatacca aattgaagat cccactcatt      540

cacagggccc tgcaactggc tcagagacca gtgtcattgc tggcctcccc ctggacctcc      600

ccaacttggc tcaaaaccaa tggggctgtc aatggtaagg gctctcttaa ggggcagcct      660

ggagacattt accatcagac ctgggccagg tattttgtga agttcctgga tgcttatgct      720

gagcacaaat tgcaattttg ggctgttaca gctgagaatg aaccctctgc aggactgctg      780

tctggctatc ctttccagtg cctgggcttt acccctgagc atcagaggga tttcattgcc      840

agggacctgg gacctactct tgccaatagc acacaccata atgtgaggct tctgatgctt      900

gatgaccaga gacttctgct gccacactgg gccaaggttg tcctgacaga tcctgaggct      960

gccaagtatg ttcatgggat tgctgtgcac tggtatctgg acttccttgc tccagctaag     1020

gccaccctgg gagaaacaca caggttgttt cccaatacaa tgctttttgc atcagaggcc     1080

tgtgtgggca gtaaattttg ggagcagtct gttaggctgg ggagctggga tagaggaatg     1140

caatactccc attctatcat caccaatctg ctctaccatg tggtggggtg gactgactgg     1200

aaccttgccc ttaaccctga gggtggcccc aattgggtca ggaattttgt ggatagtccc     1260

atcattgtgg atatcaccaa ggacacattc tataagcaac caatgttcta tcacctgggt     1320

cactttagta agtttatccc tgaggggtcc cagagggtgg gactggtggc ttcccagaag     1380

aatgatctgg atgctgtggc cctgatgcac cctgatggca gtgctgtggt tgttgttctc     1440

aatagaagct ctaaagatgt gcccttgacc atcaaagatc cagctgtggg atttctggaa     1500

acaatttccc ctggttatag catccacact tacctttgga gaaggcagtg a              1551


<210>  2
<211>  388
<212>  DNA
<213>  Homo sapiens

<400>  2
attcctgctg ggaaaagcaa gtggaggtgc tccttgaaga aacaggggga tcccaccgat       60

ctcaggggtt ctgttctggc ctgcggccct ggatcgtcca gcctgggtcg gggtggggag      120

cagacctcgc ccttatcggc tggggctgag ggtgagggtc ccgtttcccc aaaggcctag      180

cctggggttc cagccacaag ccctaccggg cagcgcccgg ccccgcccct ccaggcctgg      240

cactcgtcct caaccaagat ggcgcggatg gcttcaggcg catcacgaca ccggcgcgtc      300

acgcgacccg ccctacgggc acctcccgcg cttttcttag cgccgcagac ggtggccgag      360

cgggggaccg ggaagcatgg cccgggct                                         388


<210>  3
<211>  208
<212>  DNA
<213>  Bos taurus

<400>  3
gccctgtgcc ttctagttgc cagccatctg ttgtttgccc ctcccccgtg ccttccttga       60

ccctggaagg tgccactccc actgtccttt cctaataaaa tgaggaaatt gcatcgcatt      120

gtctgagtag gtgtcattct attctggggg gtggggtggg gcaggacagc aagggggagg      180

attgggaaga caatagcagg catgcact                                         208


<210>  4
<211>  5295
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Recombinant Construct

<400>  4
tcgcgcgttt cggtgatgac ggtgaaaacc tctgacacat gcagctcccg gagacggtca       60

cagcttgtct gtaagcggat gccgggagca gacaagcccg tcagggcgcg tcagcgggtg      120

ttggcgggtg tcggggctgg cttaactatg cggcatcaga gcagattgta ctgagagtgc      180

accatatgcg gtgtgaaata ccgcacagat gcgtaaggag aaaataccgc atcaggcgcc      240

attcgccatt caggctgcgc aactgttggg aagggcgatc ggtgcgggcc tcttcgctat      300

tacgccagct ggcgaaaggg ggatgtgctg caaggcgatt aagttgggta acgccagggt      360

tttcccagtc acgacgttgt aaaacgacgg ccagtgaatt cgagctcggt acctcgcgaa      420

tgcatctaga ggccactccc tctctgcgcg ctcgctcgct cactgaggcc gggcgaccaa      480

aggtcgcccg acgcccgggc tttgcccggg cggcctcagt gagcgagcga gcgcgcagag      540

agggagtggc caactccatc actaggggtt cctggagggg tggagtcgtg acagatctga      600

attcctgctg ggaaaagcaa gtggaggtgc tccttgaaga aacaggggga tcccaccgat      660

ctcaggggtt ctgttctggc ctgcggccct ggatcgtcca gcctgggtcg gggtggggag      720

cagacctcgc ccttatcggc tggggctgag ggtgagggtc ccgtttcccc aaaggcctag      780

cctggggttc cagccacaag ccctaccggg cagcgcccgg ccccgcccct ccaggcctgg      840

cactcgtcct caaccaagat ggcgcggatg gcttcaggcg catcacgaca ccggcgcgtc      900

acgcgacccg ccctacgggc acctcccgcg cttttcttag cgccgcagac ggtggccgag      960

cgggggaccg ggaagcatgg cccgggctgc agctctaagg taaatataaa atttttaagt     1020

gtataatgtg ttaaactact gattctaatt gtttctctct tttagattcc aacctttgga     1080

actcaattca gccaccatgg ctggcagtct tacaggtctc ctgctcctgc aagctgtctc     1140

ttgggcttct ggggccaggc cctgtatccc caaatccttt ggatactcat ctgtggtgtg     1200

tgtttgtaat gccacttatt gtgatagctt tgaccccccc acctttcctg cactgggcac     1260

cttttcaagg tatgaatcta ccaggtctgg gaggaggatg gagctgagta tggggcccat     1320

ccaagcaaac catactggca ctggcttgct gctgacactg caacctgaac agaagttcca     1380

gaaagtgaag ggctttggag gagccatgac tgatgctgct gccctcaata ttttggccct     1440

gagcccccct gctcagaatc tccttttgaa atcatacttc tctgaggagg gaattggata     1500

caatatcatc agggtgccaa tggcctcatg tgactttagt attaggactt acacctatgc     1560

tgatacccct gatgatttcc agctgcataa cttctcattg cctgaggagg ataccaaatt     1620

gaagatccca ctcattcaca gggccctgca actggctcag agaccagtgt cattgctggc     1680

ctccccctgg acctccccaa cttggctcaa aaccaatggg gctgtcaatg gtaagggctc     1740

tcttaagggg cagcctggag acatttacca tcagacctgg gccaggtatt ttgtgaagtt     1800

cctggatgct tatgctgagc acaaattgca attttgggct gttacagctg agaatgaacc     1860

ctctgcagga ctgctgtctg gctatccttt ccagtgcctg ggctttaccc ctgagcatca     1920

gagggatttc attgccaggg acctgggacc tactcttgcc aatagcacac accataatgt     1980

gaggcttctg atgcttgatg accagagact tctgctgcca cactgggcca aggttgtcct     2040

gacagatcct gaggctgcca agtatgttca tgggattgct gtgcactggt atctggactt     2100

ccttgctcca gctaaggcca ccctgggaga aacacacagg ttgtttccca atacaatgct     2160

ttttgcatca gaggcctgtg tgggcagtaa attttgggag cagtctgtta ggctggggag     2220

ctgggataga ggaatgcaat actcccattc tatcatcacc aatctgctct accatgtggt     2280

ggggtggact gactggaacc ttgcccttaa ccctgagggt ggccccaatt gggtcaggaa     2340

ttttgtggat agtcccatca ttgtggatat caccaaggac acattctata agcaaccaat     2400

gttctatcac ctgggtcact ttagtaagtt tatccctgag gggtcccaga gggtgggact     2460

ggtggcttcc cagaagaatg atctggatgc tgtggccctg atgcaccctg atggcagtgc     2520

tgtggttgtt gttctcaata gaagctctaa agatgtgccc ttgaccatca aagatccagc     2580

tgtgggattt ctggaaacaa tttcccctgg ttatagcatc cacacttacc tttggagaag     2640

gcagtgaaaa tgaaggcctg ataattgcac caccaggcct gataggccct gtgccttcta     2700

gttgccagcc atctgttgtt tgcccctccc ccgtgccttc cttgaccctg gaaggtgcca     2760

ctcccactgt cctttcctaa taaaatgagg aaattgcatc gcattgtctg agtaggtgtc     2820

attctattct ggggggtggg gtggggcagg acagcaaggg ggaggattgg gaagacaata     2880

gcaggcatgc actagtccac tccctctctg cgcgctcgct cgctcactga ggccgggcga     2940

ccaaaggtcg cccgacgccc gggctttgcc cgggcggcct cagtgagcga gcgagcgcgc     3000

agagagggat ctagatatcg gatcccgggc ccgtcgactg cagaggcctg catgcaagct     3060

tggcgtaatc atggtcatag ctgtttcctg tgtgaaattg ttatccgctc acaattccac     3120

acaacatacg agccggaagc ataaagtgta aagcctgggg tgcctaatga gtgagctaac     3180

tcacattaat tgcgttgcgc tcactgcccg ctttccagtc gggaaacctg tcgtgccagc     3240

tgcattaatg aatcggccaa cgcgcgggga gaggcggttt gcgtattggg cgctcttccg     3300

cttcctcgct cactgactcg ctgcgctcgg tcgttcggct gcggcgagcg gtatcagctc     3360

actcaaaggc ggtaatacgg ttatccacag aatcagggga taacgcagga aagaacatgt     3420

gagcaaaagg ccagcaaaag gccaggaacc gtaaaaaggc cgcgttgctg gcgtttttcc     3480

ataggctccg cccccctgac gagcatcaca aaaatcgacg ctcaagtcag aggtggcgaa     3540

acccgacagg actataaaga taccaggcgt ttccccctgg aagctccctc gtgcgctctc     3600

ctgttccgac cctgccgctt accggatacc tgtccgcctt tctcccttcg ggaagcgtgg     3660

cgctttctca tagctcacgc tgtaggtatc tcagttcggt gtaggtcgtt cgctccaagc     3720

tgggctgtgt gcacgaaccc cccgttcagc ccgaccgctg cgccttatcc ggtaactatc     3780

gtcttgagtc caacccggta agacacgact tatcgccact ggcagcagcc actggtaaca     3840

ggattagcag agcgaggtat gtaggcggtg ctacagagtt cttgaagtgg tggcctaact     3900

acggctacac tagaagaaca gtatttggta tctgcgctct gctgaagcca gttaccttcg     3960

gaaaaagagt tggtagctct tgatccggca aacaaaccac cgctggtagc ggtggttttt     4020

ttgtttgcaa gcagcagatt acgcgcagaa aaaaaggatc tcaagaagat cctttgatct     4080

tttctacggg gtctgacgct cagtggaacg aaaactcacg ttaagggatt ttggtcatga     4140

gattatcaaa aaggatcttc acctagatcc ttttaaatta aaaatgaagt tttaaatcaa     4200

tctaaagtat atatgagtaa acttggtctg acagttacca atgcttaatc agtgaggcac     4260

ctatctcagc gatctgtcta tttcgttcat ccatagttgc ctgactcccc gtcgtgtaga     4320

taactacgat acgggagggc ttaccatctg gccccagtgc tgcaatgata ccgcgagacc     4380

cacgctcacc ggctccagat ttatcagcaa taaaccagcc agccggaagg gccgagcgca     4440

gaagtggtcc tgcaacttta tccgcctcca tccagtctat taattgttgc cgggaagcta     4500

gagtaagtag ttcgccagtt aatagtttgc gcaacgttgt tgccattgct acaggcatcg     4560

tggtgtcacg ctcgtcgttt ggtatggctt cattcagctc cggttcccaa cgatcaaggc     4620

gagttacatg atcccccatg ttgtgcaaaa aagcggttag ctccttcggt cctccgatcg     4680

ttgtcagaag taagttggcc gcagtgttat cactcatggt tatggcagca ctgcataatt     4740

ctcttactgt catgccatcc gtaagatgct tttctgtgac tggtgagtac tcaaccaagt     4800

cattctgaga atagtgtatg cggcgaccga gttgctcttg cccggcgtca atacgggata     4860

ataccgcgcc acatagcaga actttaaaag tgctcatcat tggaaaacgt tcttcggggc     4920

gaaaactctc aaggatctta ccgctgttga gatccagttc gatgtaaccc actcgtgcac     4980

ccaactgatc ttcagcatct tttactttca ccagcgtttc tgggtgagca aaaacaggaa     5040

ggcaaaatgc cgcaaaaaag ggaataaggg cgacacggaa atgttgaata ctcatactct     5100

tcctttttca atattattga agcatttatc agggttattg tctcatgagc ggatacatat     5160

ttgaatgtat ttagaaaaat aaacaaatag gggttccgcg cacatttccc cgaaaagtgc     5220

cacctgacgt ctaagaaacc attattatca tgacattaac ctataaaaat aggcgtatca     5280

cgaggccctt tcgtc                                                      5295


<210>  5
<211>  497
<212>  PRT
<213>  Homo sapiens

<400>  5

Ala Arg Pro Cys Ile Pro Lys Ser Phe Gly Tyr Ser Ser Val Val Cys 
1               5                   10                  15      


Val Cys Asn Ala Thr Tyr Cys Asp Ser Phe Asp Pro Pro Thr Phe Pro 
            20                  25                  30          


Ala Leu Gly Thr Phe Ser Arg Tyr Glu Ser Thr Arg Ser Gly Arg Arg 
        35                  40                  45              


Met Glu Leu Ser Met Gly Pro Ile Gln Ala Asn His Thr Gly Thr Gly 
    50                  55                  60                  


Leu Leu Leu Thr Leu Gln Pro Glu Gln Lys Phe Gln Lys Val Lys Gly 
65                  70                  75                  80  


Phe Gly Gly Ala Met Thr Asp Ala Ala Ala Leu Asn Ile Leu Ala Leu 
                85                  90                  95      


Ser Pro Pro Ala Gln Asn Leu Leu Leu Lys Ser Tyr Phe Ser Glu Glu 
            100                 105                 110         


Gly Ile Gly Tyr Asn Ile Ile Arg Val Pro Met Ala Ser Cys Asp Phe 
        115                 120                 125             


Ser Ile Arg Thr Tyr Thr Tyr Ala Asp Thr Pro Asp Asp Phe Gln Leu 
    130                 135                 140                 


His Asn Phe Ser Leu Pro Glu Glu Asp Thr Lys Leu Lys Ile Pro Leu 
145                 150                 155                 160 


Ile His Arg Ala Leu Gln Leu Ala Gln Arg Pro Val Ser Leu Leu Ala 
                165                 170                 175     


Ser Pro Trp Thr Ser Pro Thr Trp Leu Lys Thr Asn Gly Ala Val Asn 
            180                 185                 190         


Gly Lys Gly Ser Leu Lys Gly Gln Pro Gly Asp Ile Tyr His Gln Thr 
        195                 200                 205             


Trp Ala Arg Tyr Phe Val Lys Phe Leu Asp Ala Tyr Ala Glu His Lys 
    210                 215                 220                 


Leu Gln Phe Trp Ala Val Thr Ala Glu Asn Glu Pro Ser Ala Gly Leu 
225                 230                 235                 240 


Leu Ser Gly Tyr Pro Phe Gln Cys Leu Gly Phe Thr Pro Glu His Gln 
                245                 250                 255     


Arg Asp Phe Ile Ala Arg Asp Leu Gly Pro Thr Leu Ala Asn Ser Thr 
            260                 265                 270         


His His Asn Val Arg Leu Leu Met Leu Asp Asp Gln Arg Leu Leu Leu 
        275                 280                 285             


Pro His Trp Ala Lys Val Val Leu Thr Asp Pro Glu Ala Ala Lys Tyr 
    290                 295                 300                 


Val His Gly Ile Ala Val His Trp Tyr Leu Asp Phe Leu Ala Pro Ala 
305                 310                 315                 320 


Lys Ala Thr Leu Gly Glu Thr His Arg Leu Phe Pro Asn Thr Met Leu 
                325                 330                 335     


Phe Ala Ser Glu Ala Cys Val Gly Ser Lys Phe Trp Glu Gln Ser Val 
            340                 345                 350         


Arg Leu Gly Ser Trp Asp Arg Gly Met Gln Tyr Ser His Ser Ile Ile 
        355                 360                 365             


Thr Asn Leu Leu Tyr His Val Val Gly Trp Thr Asp Trp Asn Leu Ala 
    370                 375                 380                 


Leu Asn Pro Glu Gly Gly Pro Asn Trp Val Arg Asn Phe Val Asp Ser 
385                 390                 395                 400 


Pro Ile Ile Val Asp Ile Thr Lys Asp Thr Phe Tyr Lys Gln Pro Met 
                405                 410                 415     


Phe Tyr His Leu Gly His Phe Ser Lys Phe Ile Pro Glu Gly Ser Gln 
            420                 425                 430         


Arg Val Gly Leu Val Ala Ser Gln Lys Asn Asp Leu Asp Ala Val Ala 
        435                 440                 445             


Leu Met His Pro Asp Gly Ser Ala Val Val Val Val Leu Asn Arg Ser 
    450                 455                 460                 


Ser Lys Asp Val Pro Leu Thr Ile Lys Asp Pro Ala Val Gly Phe Leu 
465                 470                 475                 480 


Glu Thr Ile Ser Pro Gly Tyr Ser Ile His Thr Tyr Leu Trp Arg Arg 
                485                 490                 495     


Gln 
    


<210>  6
<211>  516
<212>  PRT
<213>  Homo sapiens

<400>  6

Met Ala Gly Ser Leu Thr Gly Leu Leu Leu Leu Gln Ala Val Ser Trp 
1               5                   10                  15      


Ala Ser Gly Ala Arg Pro Cys Ile Pro Lys Ser Phe Gly Tyr Ser Ser 
            20                  25                  30          


Val Val Cys Val Cys Asn Ala Thr Tyr Cys Asp Ser Phe Asp Pro Pro 
        35                  40                  45              


Thr Phe Pro Ala Leu Gly Thr Phe Ser Arg Tyr Glu Ser Thr Arg Ser 
    50                  55                  60                  


Gly Arg Arg Met Glu Leu Ser Met Gly Pro Ile Gln Ala Asn His Thr 
65                  70                  75                  80  


Gly Thr Gly Leu Leu Leu Thr Leu Gln Pro Glu Gln Lys Phe Gln Lys 
                85                  90                  95      


Val Lys Gly Phe Gly Gly Ala Met Thr Asp Ala Ala Ala Leu Asn Ile 
            100                 105                 110         


Leu Ala Leu Ser Pro Pro Ala Gln Asn Leu Leu Leu Lys Ser Tyr Phe 
        115                 120                 125             


Ser Glu Glu Gly Ile Gly Tyr Asn Ile Ile Arg Val Pro Met Ala Ser 
    130                 135                 140                 


Cys Asp Phe Ser Ile Arg Thr Tyr Thr Tyr Ala Asp Thr Pro Asp Asp 
145                 150                 155                 160 


Phe Gln Leu His Asn Phe Ser Leu Pro Glu Glu Asp Thr Lys Leu Lys 
                165                 170                 175     


Ile Pro Leu Ile His Arg Ala Leu Gln Leu Ala Gln Arg Pro Val Ser 
            180                 185                 190         


Leu Leu Ala Ser Pro Trp Thr Ser Pro Thr Trp Leu Lys Thr Asn Gly 
        195                 200                 205             


Ala Val Asn Gly Lys Gly Ser Leu Lys Gly Gln Pro Gly Asp Ile Tyr 
    210                 215                 220                 


His Gln Thr Trp Ala Arg Tyr Phe Val Lys Phe Leu Asp Ala Tyr Ala 
225                 230                 235                 240 


Glu His Lys Leu Gln Phe Trp Ala Val Thr Ala Glu Asn Glu Pro Ser 
                245                 250                 255     


Ala Gly Leu Leu Ser Gly Tyr Pro Phe Gln Cys Leu Gly Phe Thr Pro 
            260                 265                 270         


Glu His Gln Arg Asp Phe Ile Ala Arg Asp Leu Gly Pro Thr Leu Ala 
        275                 280                 285             


Asn Ser Thr His His Asn Val Arg Leu Leu Met Leu Asp Asp Gln Arg 
    290                 295                 300                 


Leu Leu Leu Pro His Trp Ala Lys Val Val Leu Thr Asp Pro Glu Ala 
305                 310                 315                 320 


Ala Lys Tyr Val His Gly Ile Ala Val His Trp Tyr Leu Asp Phe Leu 
                325                 330                 335     


Ala Pro Ala Lys Ala Thr Leu Gly Glu Thr His Arg Leu Phe Pro Asn 
            340                 345                 350         


Thr Met Leu Phe Ala Ser Glu Ala Cys Val Gly Ser Lys Phe Trp Glu 
        355                 360                 365             


Gln Ser Val Arg Leu Gly Ser Trp Asp Arg Gly Met Gln Tyr Ser His 
    370                 375                 380                 


Ser Ile Ile Thr Asn Leu Leu Tyr His Val Val Gly Trp Thr Asp Trp 
385                 390                 395                 400 


Asn Leu Ala Leu Asn Pro Glu Gly Gly Pro Asn Trp Val Arg Asn Phe 
                405                 410                 415     


Val Asp Ser Pro Ile Ile Val Asp Ile Thr Lys Asp Thr Phe Tyr Lys 
            420                 425                 430         


Gln Pro Met Phe Tyr His Leu Gly His Phe Ser Lys Phe Ile Pro Glu 
        435                 440                 445             


Gly Ser Gln Arg Val Gly Leu Val Ala Ser Gln Lys Asn Asp Leu Asp 
    450                 455                 460                 


Ala Val Ala Leu Met His Pro Asp Gly Ser Ala Val Val Val Val Leu 
465                 470                 475                 480 


Asn Arg Ser Ser Lys Asp Val Pro Leu Thr Ile Lys Asp Pro Ala Val 
                485                 490                 495     


Gly Phe Leu Glu Thr Ile Ser Pro Gly Tyr Ser Ile His Thr Tyr Leu 
            500                 505                 510         


Trp Arg Arg Gln 
        515     


<210>  7
<211>  1611
<212>  DNA
<213>  Homo sapiens

<400>  7
atggagtttt caagtccttc cagagaggaa tgtcccaagc ctttgagtag ggtaagcatc       60

atggctggca gcctcacagg tttgcttcta cttcaggcag tgtcgtgggc atcaggtgcc      120

cgcccctgca tccctaaaag cttcggctac agctcggtgg tgtgtgtctg caatgccaca      180

tactgtgact cctttgaccc cccgaccttt cctgcccttg gtaccttcag ccgctatgag      240

agtacacgca gtgggcgacg gatggagctg agtatggggc ccatccaggc taatcacacg      300

ggcacaggcc tgctactgac cctgcagcca gaacagaagt tccagaaagt gaagggattt      360

ggaggggcca tgacagatgc tgctgctctc aacatccttg ccctgtcacc ccctgcccaa      420

aatttgctac ttaaatcgta cttctctgaa gaaggaatcg gatataacat catccgggta      480

cccatggcca gctgtgactt ctccatccgc acctacacct atgcagacac ccctgatgat      540

ttccagttgc acaacttcag cctcccagag gaagatacca agctcaagat acccctgatt      600

caccgagccc tgcagttggc ccagcgtccc gtttcactcc ttgccagccc ctggacatca      660

cccacttggc tcaagaccaa tggagcggtg aatgggaagg ggtcactcaa gggacagccc      720

ggagacatct accaccagac ctgggccaga tactttgtga agttcctgga tgcctatgct      780

gagcacaagt tacagttctg ggcagtgaca gctgaaaatg agccttctgc tgggctgttg      840

agtggatacc ccttccagtg cctgggcttc acccctgaac atcagcgaga cttcattgcc      900

cgtgacctag gtcctaccct cgccaacagt actcaccaca atgtccgcct actcatgctg      960

gatgaccaac gcttgctgct gccccactgg gcaaaggtgg tactgacaga cccagaagca     1020

gctaaatatg ttcatggcat tgctgtacat tggtacctgg actttctggc tccagccaaa     1080

gccaccctag gggagacaca ccgcctgttc cccaacacca tgctctttgc ctcagaggcc     1140

tgtgtgggct ccaagttctg ggagcagagt gtgcggctag gctcctggga tcgagggatg     1200

cagtacagcc acagcatcat cacgaacctc ctgtaccatg tggtcggctg gaccgactgg     1260

aaccttgccc tgaaccccga aggaggaccc aattgggtgc gtaactttgt cgacagtccc     1320

atcattgtag acatcaccaa ggacacgttt tacaaacagc ccatgttcta ccaccttggc     1380

cacttcagca agttcattcc tgagggctcc cagagagtgg ggctggttgc cagtcagaag     1440

aacgacctgg acgcagtggc actgatgcat cccgatggct ctgctgttgt ggtcgtgcta     1500

aaccgctcct ctaaggatgt gcctcttacc atcaaggatc ctgctgtggg cttcctggag     1560

acaatctcac ctggctactc cattcacacc tacctgtggc atcgccagtg a              1611


<210>  8
<211>  536
<212>  PRT
<213>  Homo sapiens

<400>  8

Met Glu Phe Ser Ser Pro Ser Arg Glu Glu Cys Pro Lys Pro Leu Ser 
1               5                   10                  15      


Arg Val Ser Ile Met Ala Gly Ser Leu Thr Gly Leu Leu Leu Leu Gln 
            20                  25                  30          


Ala Val Ser Trp Ala Ser Gly Ala Arg Pro Cys Ile Pro Lys Ser Phe 
        35                  40                  45              


Gly Tyr Ser Ser Val Val Cys Val Cys Asn Ala Thr Tyr Cys Asp Ser 
    50                  55                  60                  


Phe Asp Pro Pro Thr Phe Pro Ala Leu Gly Thr Phe Ser Arg Tyr Glu 
65                  70                  75                  80  


Ser Thr Arg Ser Gly Arg Arg Met Glu Leu Ser Met Gly Pro Ile Gln 
                85                  90                  95      


Ala Asn His Thr Gly Thr Gly Leu Leu Leu Thr Leu Gln Pro Glu Gln 
            100                 105                 110         


Lys Phe Gln Lys Val Lys Gly Phe Gly Gly Ala Met Thr Asp Ala Ala 
        115                 120                 125             


Ala Leu Asn Ile Leu Ala Leu Ser Pro Pro Ala Gln Asn Leu Leu Leu 
    130                 135                 140                 


Lys Ser Tyr Phe Ser Glu Glu Gly Ile Gly Tyr Asn Ile Ile Arg Val 
145                 150                 155                 160 


Pro Met Ala Ser Cys Asp Phe Ser Ile Arg Thr Tyr Thr Tyr Ala Asp 
                165                 170                 175     


Thr Pro Asp Asp Phe Gln Leu His Asn Phe Ser Leu Pro Glu Glu Asp 
            180                 185                 190         


Thr Lys Leu Lys Ile Pro Leu Ile His Arg Ala Leu Gln Leu Ala Gln 
        195                 200                 205             


Arg Pro Val Ser Leu Leu Ala Ser Pro Trp Thr Ser Pro Thr Trp Leu 
    210                 215                 220                 


Lys Thr Asn Gly Ala Val Asn Gly Lys Gly Ser Leu Lys Gly Gln Pro 
225                 230                 235                 240 


Gly Asp Ile Tyr His Gln Thr Trp Ala Arg Tyr Phe Val Lys Phe Leu 
                245                 250                 255     


Asp Ala Tyr Ala Glu His Lys Leu Gln Phe Trp Ala Val Thr Ala Glu 
            260                 265                 270         


Asn Glu Pro Ser Ala Gly Leu Leu Ser Gly Tyr Pro Phe Gln Cys Leu 
        275                 280                 285             


Gly Phe Thr Pro Glu His Gln Arg Asp Phe Ile Ala Arg Asp Leu Gly 
    290                 295                 300                 


Pro Thr Leu Ala Asn Ser Thr His His Asn Val Arg Leu Leu Met Leu 
305                 310                 315                 320 


Asp Asp Gln Arg Leu Leu Leu Pro His Trp Ala Lys Val Val Leu Thr 
                325                 330                 335     


Asp Pro Glu Ala Ala Lys Tyr Val His Gly Ile Ala Val His Trp Tyr 
            340                 345                 350         


Leu Asp Phe Leu Ala Pro Ala Lys Ala Thr Leu Gly Glu Thr His Arg 
        355                 360                 365             


Leu Phe Pro Asn Thr Met Leu Phe Ala Ser Glu Ala Cys Val Gly Ser 
    370                 375                 380                 


Lys Phe Trp Glu Gln Ser Val Arg Leu Gly Ser Trp Asp Arg Gly Met 
385                 390                 395                 400 


Gln Tyr Ser His Ser Ile Ile Thr Asn Leu Leu Tyr His Val Val Gly 
                405                 410                 415     


Trp Thr Asp Trp Asn Leu Ala Leu Asn Pro Glu Gly Gly Pro Asn Trp 
            420                 425                 430         


Val Arg Asn Phe Val Asp Ser Pro Ile Ile Val Asp Ile Thr Lys Asp 
        435                 440                 445             


Thr Phe Tyr Lys Gln Pro Met Phe Tyr His Leu Gly His Phe Ser Lys 
    450                 455                 460                 


Phe Ile Pro Glu Gly Ser Gln Arg Val Gly Leu Val Ala Ser Gln Lys 
465                 470                 475                 480 


Asn Asp Leu Asp Ala Val Ala Leu Met His Pro Asp Gly Ser Ala Val 
                485                 490                 495     


Val Val Val Leu Asn Arg Ser Ser Lys Asp Val Pro Leu Thr Ile Lys 
            500                 505                 510         


Asp Pro Ala Val Gly Phe Leu Glu Thr Ile Ser Pro Gly Tyr Ser Ile 
        515                 520                 525             


His Thr Tyr Leu Trp Arg Arg Gln 
    530                 535     


<210>  9
<211>  644
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Recombinant construct

<400>  9
ctagttatta atagtaatca attacggggt cattagttca tagcccatat atggagttcc       60

gcgttacata acttacggta aatggcccgc ctggctgacc gcccaacgac ccccgcccat      120

tgacgtcaat aatgacgtat gttcccatag taacgccaat agggactttc cattgacgtc      180

aatgggtgga gtatttacgg taaactgccc acttggcagt acatcaagtg tatcatatgc      240

caagtacgcc ccctattgac gtcaatgacg gtaaatggcc cgcctggcat tatgcccagt      300

acatgacctt atgggacttt cctacttggc agtacatcta cgtattagtc atcgctatta      360

ccatggtcga ggtgagcccc acgttctgct tcactctccc catctccccc ccctccccac      420

ccccaatttt gtatttattt attttttaat tattttgtgc agcgatgggg gcgggggggg      480

ggggggggcg cgcgccaggc ggggcggggc ggggcgaggg gcggggcggg gcgaggcgga      540

gaggtgcggc ggcagccaat cagagcggcg cgctccgaaa gtttcctttt atggcgaggc      600

ggcggcggcg gcggccctat aaaaagcgaa gcgcgcggcg ggcg                       644


<210>  10
<211>  720
<212>  DNA
<213>  Aequorea victoria

<400>  10
atggtgagca agggcgagga gctgttcacc ggggtggtgc ccatcctggt cgagctggac       60

ggcgacgtaa acggccacaa gttcagcgtg tccggcgagg gcgagggcga tgccacctac      120

ggcaagctga ccctgaagtt catctgcacc accggcaagc tgcccgtgcc ctggcccacc      180

ctcgtgacca ccctgaccta cggcgtgcag tgcttcagcc gctaccccga ccacatgaag      240

cagcacgact tcttcaagtc cgccatgccc gaaggctacg tccaggagcg caccatcttc      300

ttcaaggacg acggcaacta caagacccgc gccgaggtga agttcgaggg cgacaccctg      360

gtgaaccgca tcgagctgaa gggcatcgac ttcaaggagg acggcaacat cctggggcac      420

aagctggagt acaactacaa cagccacaac gtctatatca tggccgacaa gcagaagaac      480

ggcatcaagg tgaacttcaa gatccgccac aacatcgagg acggcagcgt gcagctcgcc      540

gaccactacc agcagaacac ccccatcggc gacggccccg tgctgctgcc cgacaaccac      600

tacctgagca cccagtccgc cctgagcaaa gaccccaacg agaagcgcga tcacatggtc      660

ctgctggagt tcgtgaccgc cgccgggatc actctcggca tggacgagct gtacaagtga      720


<210>  11
<211>  1611
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Human GBA1 coding nucleotide sequence (IDT optimized sequence)

<400>  11
atggagttct catctccctc acgagaagaa tgtccgaaac ctctttcaag agtaagcatc       60

atggccggca gcttgaccgg tcttttgttg ttgcaggccg tgtcctgggc ctcaggtgct      120

aggccatgca ttcctaaatc cttcggctat agtagcgtgg tttgcgtctg caacgccaca      180

tactgtgaca gtttcgatcc acctaccttc ccagcgctgg gtaccttctc acggtatgaa      240

tcaacgcgat cagggcgcag aatggaactt tcaatggggc caatccaagc taaccacacg      300

ggaacgggtc ttctgctgac gctccaaccg gaacaaaagt tccaaaaggt aaaaggcttt      360

ggaggtgcga tgactgatgc cgcagcactc aacatcctgg cgctctcacc gccggcacaa      420

aatttgctgt tgaagagtta tttctcagaa gaagggatcg gttacaacat catacgggtc      480

ccgatggcga gctgtgactt ttctataaga acatatacct atgcggatac gcccgacgat      540

ttccaacttc ataattttag tctgcctgag gaagacacaa agttgaagat accgctgata      600

cacagagcat tgcagcttgc tcaacgaccg gtcagcttgc ttgccagccc atggacaagt      660

ccaacatggc ttaagaccaa tggcgcggtt aatggcaagg gatccctgaa gggccagccg      720

ggagacatct atcatcaaac ttgggcgcgg tattttgtca agttcttgga cgcctacgct      780

gagcacaaac tgcagttctg ggccgttacc gccgaaaatg aaccatccgc cggactgctt      840

tctggctacc ctttccaatg tcttggcttt acgcctgaac accaaagaga cttcattgct      900

cgggaccttg gtccaacgct cgcgaacagt actcatcata atgtacgact cttgatgctc      960

gatgaccagc gactgttgct tccacattgg gccaaggtag ttctgaccga ccccgaagcc     1020

gctaaatacg tccacggcat tgctgtccat tggtaccttg actttttggc tcccgcaaaa     1080

gccactctgg gtgaaacaca cagactcttt ccaaacacga tgcttttcgc atcagaagcc     1140

tgcgtcggaa gtaaattttg ggaacagtca gtaaggttgg gtagttggga tcgcgggatg     1200

caatatagtc atagcattat taccaacttg ctttatcacg tcgttgggtg gacagattgg     1260

aacctcgcgt tgaatcctga aggcggccct aattgggtaa gaaactttgt tgattcacct     1320

attatcgtcg acataaccaa ggacacattc tacaagcaac cgatgttcta tcaccttggg     1380

catttcagta aattcatacc agagggcagc cagcgcgtcg ggttggtagc ctctcaaaaa     1440

aacgatttgg atgcggtcgc tctgatgcat cccgacggga gcgcagtagt cgttgtcctt     1500

aaccgaagct ccaaggatgt acccctcacg attaaggacc ctgctgtcgg gttccttgaa     1560

actataagtc ccggctatag tattcatact tatctctgga gaagacagtg a              1611


<210>  12
<211>  1611
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Human GBA1 coding nucleotide sequence (GenScript optimized 
       sequence)

<400>  12
atggagtttt caagcccctc acgggaagag tgccctaagc ccctgtcacg ggtctcaatt       60

atggccggga gcctgactgg cctgctgctg ctgcaggccg tgagctgggc atcaggagcc      120

aggccttgca tcccaaagtc tttcggctac agctccgtgg tgtgcgtgtg caacgccacc      180

tattgtgact ccttcgatcc ccctaccttt cccgccctgg gcacattttc tagatacgag      240

tctacacgca gcggccggag aatggagctg agcatgggcc ctatccaggc caatcacacc      300

ggaacaggcc tgctgctgac cctgcagcca gagcagaagt tccagaaggt gaagggcttt      360

ggaggagcaa tgacagacgc agccgccctg aacatcctgg ccctgtcccc acccgcccag      420

aatctgctgc tgaagtccta cttctctgag gagggcatcg gctataacat catccgggtg      480

cccatggcca gctgcgactt ttccatcaga acctacacat atgccgatac ccctgacgat      540

ttccagctgc acaatttttc cctgccagag gaggatacaa agctgaagat ccccctgatc      600

caccgggccc tgcagctggc acagcggccc gtgagcctgc tggccagccc ctggacctcc      660

cctacatggc tgaagaccaa cggcgccgtg aatggcaagg gctctctgaa gggacagcca      720

ggcgacatct accaccagac atgggccaga tatttcgtga agtttctgga tgcctacgcc      780

gagcacaagc tgcagttctg ggccgtgacc gcagagaacg agccttctgc cggcctgctg      840

agcggctatc ccttccagtg cctgggcttt acacctgagc accagcggga ctttatcgcc      900

agagatctgg gcccaaccct ggccaactcc acacaccaca atgtgaggct gctgatgctg      960

gacgatcagc gcctgctgct gcctcactgg gccaaggtgg tgctgaccga cccagaggcc     1020

gccaagtacg tgcacggcat cgccgtgcac tggtatctgg atttcctggc accagcaaag     1080

gccaccctgg gagagacaca ccggctgttc cctaacacca tgctgtttgc cagcgaggcc     1140

tgcgtgggct ccaagttttg ggagcagtcc gtgaggctgg gatcttggga caggggcatg     1200

cagtactccc actctatcat caccaatctg ctgtatcacg tggtgggctg gacagactgg     1260

aacctggccc tgaatccaga gggcggcccc aactgggtga gaaatttcgt ggatagcccc     1320

atcatcgtgg acatcaccaa ggatacattc tacaagcagc caatgtttta tcacctgggc     1380

cacttctcta agtttatccc agagggcagc cagagggtgg gcctggtggc cagccagaag     1440

aacgacctgg atgccgtggc cctgatgcac cctgatggct ccgccgtggt ggtggtgctg     1500

aatcgctcta gcaaggacgt gcctctgacc atcaaggatc cagccgtggg cttcctggag     1560

actatttccc ccggctattc aattcatacc tatctgtgga gaaggcagtg a              1611


<210>  13
<211>  448
<212>  DNA
<213>  homo sapiens

<400>  13
agtgcaagtg ggttttagga ccaggatgag gcggggtggg ggtgcctacc tgacgaccga       60

ccccgaccca ctggacaagc acccaacccc cattccccaa attgcgcatc ccctatcaga      120

gagggggagg ggaaacagga tgcggcgagg cgcgtgcgca ctgccagctt cagcaccgcg      180

gacagtgcct tcgcccccgc ctggcggcgc gcgccaccgc cgcctcagca ctgaaggcgc      240

gctgacgtca ctcgccggtc ccccgcaaac tccccttccc ggccaccttg gtcgcgtccg      300

cgccgccgcc ggcccagccg gaccgcacca cgcgaggcgc gagatagggg ggcacgggcg      360

cgaccatctg cgctgcggcg ccggcgactc agcgctgcct cagtctgcgg tgggcagcgg      420

aggagtcgtg tcgtgcctga gagcgcag                                         448


<210>  14
<211>  735
<212>  PRT
<213>  Artificial sequence

<220>
<223>  AAV TT capsid protein

<400>  14

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


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


Lys Pro Ala Glu Arg His Lys Asp Asp Ser 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 Glu Ala Asp Ala Ala Ala Leu Glu His Asp Lys Ala Tyr Asp 
65                  70                  75                  80  


Arg Gln Leu Asp Ser 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 Ile Leu Glu Pro 
        115                 120                 125             


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


Pro Val Glu His Ser Pro Ala Glu Pro Asp Ser Ser Ser Gly Thr Gly 
145                 150                 155                 160 


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


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


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


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


Ser Gly Asn Trp His Cys Asp Ser Thr Trp Met 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 Gln Ser Gly Ala Ser Asn Asp Asn His Tyr 
            260                 265                 270         


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


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


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


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


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


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


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


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


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


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


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


Asn Thr Pro Ser Gly Thr Thr Thr Met Ser Arg Leu Gln Phe Ser Gln 
    450                 455                 460                 


Ala Gly Ala Ser Asp Ile Arg Asp Gln Ser Arg Asn Trp Leu Pro Gly 
465                 470                 475                 480 


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


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


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


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


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


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


Gly Ser Val Ser Thr Asn Leu Gln Ser Gly Asn Thr Gln Ala Ala Thr 
            580                 585                 590         


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


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


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


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


Pro Ser Thr Thr Phe Ser Ala Ala Lys Phe Ala Ser Phe Ile Thr Gln 
            660                 665                 670         


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


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


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


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


<210>  15
<211>  145
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Flip ITR of AAV2

<400>  15
ttggccactc cctctctgcg cgctcgctcg ctcactgagg ccgggcgacc aaaggtcgcc       60

cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc gagcgcgcag agagggagtg      120

gccaactcca tcactagggg ttcct                                            145


<210>  16
<211>  145
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Flop ITR of AAV2

<400>  16
aggaacccct agtgatggag ttggccactc cctctctgcg cgctcgctcg ctcactgagg       60

ccgggcgacc aaaggtcgcc cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc      120

gagcgcgcag agagggagtg gccaa                                            145


<210>  17
<211>  449
<212>  PRT
<213>  homo sapiens

<400>  17

Met Glu Leu Ser Met Gly Pro Ile Gln Ala Asn His Thr Gly Thr Gly 
1               5                   10                  15      


Leu Leu Leu Thr Leu Gln Pro Glu Gln Lys Phe Gln Lys Val Lys Gly 
            20                  25                  30          


Phe Gly Gly Ala Met Thr Asp Ala Ala Ala Leu Asn Ile Leu Ala Leu 
        35                  40                  45              


Ser Pro Pro Ala Gln Asn Leu Leu Leu Lys Ser Tyr Phe Ser Glu Glu 
    50                  55                  60                  


Gly Ile Gly Tyr Asn Ile Ile Arg Val Pro Met Ala Ser Cys Asp Phe 
65                  70                  75                  80  


Ser Ile Arg Thr Tyr Thr Tyr Ala Asp Thr Pro Asp Asp Phe Gln Leu 
                85                  90                  95      


His Asn Phe Ser Leu Pro Glu Glu Asp Thr Lys Leu Lys Ile Pro Leu 
            100                 105                 110         


Ile His Arg Ala Leu Gln Leu Ala Gln Arg Pro Val Ser Leu Leu Ala 
        115                 120                 125             


Ser Pro Trp Thr Ser Pro Thr Trp Leu Lys Thr Asn Gly Ala Val Asn 
    130                 135                 140                 


Gly Lys Gly Ser Leu Lys Gly Gln Pro Gly Asp Ile Tyr His Gln Thr 
145                 150                 155                 160 


Trp Ala Arg Tyr Phe Val Lys Phe Leu Asp Ala Tyr Ala Glu His Lys 
                165                 170                 175     


Leu Gln Phe Trp Ala Val Thr Ala Glu Asn Glu Pro Ser Ala Gly Leu 
            180                 185                 190         


Leu Ser Gly Tyr Pro Phe Gln Cys Leu Gly Phe Thr Pro Glu His Gln 
        195                 200                 205             


Arg Asp Phe Ile Ala Arg Asp Leu Gly Pro Thr Leu Ala Asn Ser Thr 
    210                 215                 220                 


His His Asn Val Arg Leu Leu Met Leu Asp Asp Gln Arg Leu Leu Leu 
225                 230                 235                 240 


Pro His Trp Ala Lys Val Val Leu Thr Asp Pro Glu Ala Ala Lys Tyr 
                245                 250                 255     


Val His Gly Ile Ala Val His Trp Tyr Leu Asp Phe Leu Ala Pro Ala 
            260                 265                 270         


Lys Ala Thr Leu Gly Glu Thr His Arg Leu Phe Pro Asn Thr Met Leu 
        275                 280                 285             


Phe Ala Ser Glu Ala Cys Val Gly Ser Lys Phe Trp Glu Gln Ser Val 
    290                 295                 300                 


Arg Leu Gly Ser Trp Asp Arg Gly Met Gln Tyr Ser His Ser Ile Ile 
305                 310                 315                 320 


Thr Asn Leu Leu Tyr His Val Val Gly Trp Thr Asp Trp Asn Leu Ala 
                325                 330                 335     


Leu Asn Pro Glu Gly Gly Pro Asn Trp Val Arg Asn Phe Val Asp Ser 
            340                 345                 350         


Pro Ile Ile Val Asp Ile Thr Lys Asp Thr Phe Tyr Lys Gln Pro Met 
        355                 360                 365             


Phe Tyr His Leu Gly His Phe Ser Lys Phe Ile Pro Glu Gly Ser Gln 
    370                 375                 380                 


Arg Val Gly Leu Val Ala Ser Gln Lys Asn Asp Leu Asp Ala Val Ala 
385                 390                 395                 400 


Leu Met His Pro Asp Gly Ser Ala Val Val Val Val Leu Asn Arg Ser 
                405                 410                 415     


Ser Lys Asp Val Pro Leu Thr Ile Lys Asp Pro Ala Val Gly Phe Leu 
            420                 425                 430         


Glu Thr Ile Ser Pro Gly Tyr Ser Ile His Thr Tyr Leu Trp Arg Arg 
        435                 440                 445             


Gln 
    


<210>  18
<211>  449
<212>  PRT
<213>  homo sapiens

<400>  18

Met Glu Leu Ser Met Gly Pro Ile Gln Ala Asn His Thr Gly Thr Gly 
1               5                   10                  15      


Leu Leu Leu Thr Leu Gln Pro Glu Gln Lys Phe Gln Lys Val Lys Gly 
            20                  25                  30          


Phe Gly Gly Ala Met Thr Asp Ala Ala Ala Leu Asn Ile Leu Ala Leu 
        35                  40                  45              


Ser Pro Pro Ala Gln Asn Leu Leu Leu Lys Ser Tyr Phe Ser Glu Glu 
    50                  55                  60                  


Gly Ile Gly Tyr Asn Ile Ile Arg Val Pro Met Ala Ser Cys Asp Phe 
65                  70                  75                  80  


Ser Ile Arg Thr Tyr Thr Tyr Ala Asp Thr Pro Asp Asp Phe Gln Leu 
                85                  90                  95      


His Asn Phe Ser Leu Pro Glu Glu Asp Thr Lys Leu Lys Ile Pro Leu 
            100                 105                 110         


Ile His Arg Ala Leu Gln Leu Ala Gln Arg Pro Val Ser Leu Leu Ala 
        115                 120                 125             


Ser Pro Trp Thr Ser Pro Thr Trp Leu Lys Thr Asn Gly Ala Val Asn 
    130                 135                 140                 


Gly Lys Gly Ser Leu Lys Gly Gln Pro Gly Asp Ile Tyr His Gln Thr 
145                 150                 155                 160 


Trp Ala Arg Tyr Phe Val Lys Phe Leu Asp Ala Tyr Ala Glu His Lys 
                165                 170                 175     


Leu Gln Phe Trp Ala Val Thr Ala Glu Asn Glu Pro Ser Ala Gly Leu 
            180                 185                 190         


Leu Ser Gly Tyr Pro Phe Gln Cys Leu Gly Phe Thr Pro Glu His Gln 
        195                 200                 205             


Arg Asp Phe Ile Ala Arg Asp Leu Gly Pro Thr Leu Ala Asn Ser Thr 
    210                 215                 220                 


His His Asn Val Arg Leu Leu Met Leu Asp Asp Gln Arg Leu Leu Leu 
225                 230                 235                 240 


Pro His Trp Ala Lys Val Val Leu Thr Asp Pro Glu Ala Ala Lys Tyr 
                245                 250                 255     


Val His Gly Ile Ala Val His Trp Tyr Leu Asp Phe Leu Ala Pro Ala 
            260                 265                 270         


Lys Ala Thr Leu Gly Glu Thr His Arg Leu Phe Pro Asn Thr Met Leu 
        275                 280                 285             


Phe Ala Ser Glu Ala Cys Val Gly Ser Lys Phe Trp Glu Gln Ser Val 
    290                 295                 300                 


Arg Leu Gly Ser Trp Asp Arg Gly Met Gln Tyr Ser His Ser Ile Ile 
305                 310                 315                 320 


Thr Asn Leu Leu Tyr His Val Val Gly Trp Thr Asp Trp Asn Leu Ala 
                325                 330                 335     


Leu Asn Pro Glu Gly Gly Pro Asn Trp Val Arg Asn Phe Val Asp Ser 
            340                 345                 350         


Pro Ile Ile Val Asp Ile Thr Lys Asp Thr Phe Tyr Lys Gln Pro Met 
        355                 360                 365             


Phe Tyr His Leu Gly His Phe Ser Lys Phe Ile Pro Glu Gly Ser Gln 
    370                 375                 380                 


Arg Val Gly Leu Val Ala Ser Gln Lys Asn Asp Leu Asp Ala Val Ala 
385                 390                 395                 400 


Leu Met His Pro Asp Gly Ser Ala Val Val Val Val Leu Asn Arg Ser 
                405                 410                 415     


Ser Lys Asp Val Pro Leu Thr Ile Lys Asp Pro Ala Val Gly Phe Leu 
            420                 425                 430         


Glu Thr Ile Ser Pro Gly Tyr Ser Ile His Thr Tyr Leu Trp Arg Arg 
        435                 440                 445             


Gln 
    


<210>  19
<211>  1611
<212>  DNA
<213>  Artificial sequence

<220>
<223>  human GBA1 gene variant

<400>  19
atggagtttt caagtccttc cagagaggaa tgtcccaagc ctttgagtag ggtaagcatc       60

atggctggca gcctcacagg attgcttcta cttcaggcag tgtcgtgggc atcaggtgcc      120

cgcccctgca tccctaaaag cttcggctac agctcggtgg tgtgtgtctg caatgccaca      180

tactgtgact cctttgaccc cccgaccttt cctgcccttg gtaccttcag ccgctatgag      240

agtacacgca gtgggcgacg gatggagctg agtatggggc ccatccaggc taatcacacg      300

ggcacaggcc tgctactgac cctgcagcca gaacagaagt tccagaaagt gaagggattt      360

ggaggggcca tgacagatgc tgctgctctc aacatccttg ccctgtcacc ccctgcccaa      420

aatttgctac ttaaatcgta cttctctgaa gaaggaatcg gatataacat catccgggta      480

cccatggcca gctgtgactt ctccatccgc acctacacct atgcagacac ccctgatgat      540

ttccagttgc acaacttcag cctcccagag gaagatacca agctcaagat acccctgatt      600

caccgagccc tgcagttggc ccagcgtccc gtttcactcc ttgccagccc ctggacatca      660

cccacttggc tcaagaccaa tggagcggtg aatgggaagg ggtcactcaa gggacagccc      720

ggagacatct accaccagac ctgggccaga tactttgtga agttcctgga tgcctatgct      780

gagcacaagt tacagttctg ggcagtgaca gctgaaaatg agccttctgc tgggctgttg      840

agtggatacc ccttccagtg cctgggcttc acccctgaac atcagcgaga cttcattgcc      900

cgtgacctag gtcctaccct cgccaacagt actcaccaca atgtccgcct actcatgctg      960

gatgaccaac gcttgctgct gccccactgg gcaaaggtgg tactgacaga cccagaagca     1020

gctaaatatg ttcatggcat tgctgtacat tggtacctgg actttctggc tccagccaaa     1080

gccaccctag gggagacaca ccgcctgttc cccaacacca tgctctttgc ctcagaggcc     1140

tgtgtgggct ccaagttctg ggagcagagt gtgcggctag gctcctggga tcgagggatg     1200

cagtacagcc acagcatcat cacgaacctc ctgtaccatg tggtcggctg gaccgactgg     1260

aaccttgccc tgaaccccga aggaggaccc aattgggtgc gtaactttgt cgacagtccc     1320

atcattgtag acatcaccaa ggacacgttt tacaaacagc ccatgttcta ccaccttggc     1380

cacttcagca agttcattcc tgagggctcc cagagagtgg ggctggttgc cagtcagaag     1440

aacgacctgg acgcagtggc actgatgcat cccgatggct ctgctgttgt ggtcgtgcta     1500

aaccgctcct ctaaggatgt gcctcttacc atcaaggatc ctgctgtggg cttcctggag     1560

acaatctcac ctggctactc cattcacacc tacctgtggc gtcgccagtg a              1611


<210>  20
<211>  376
<212>  DNA
<213>  Artificial sequence

<220>
<223>  nGUSB promoter #2

<400>  20
attcctgctg ggaaaagcaa gtggaggtgc tccttgaaga aacaggggga tcccaccgat       60

ctcaggggtt ctgttctggc ctgcggccct ggatcgtcca gcctgggtcg gggtggggag      120

cagacctcgc ccttatcggc tggggctgag ggtgagggtc ccgtttcccc aaaggcctag      180

cctggggttc cagccacaag ccctaccggg cagcgcccgg ccccgcccct ccaggcctgg      240

cactcgtcct caaccaagat ggcgcggatg gcttcaggcg catcacgaca ccggcgcgtc      300

acgcgacccg ccctacgggc acctcccgcg cttttcttag cgccgcagac ggtggccgag      360

cgggggaccg ggaagc                                                      376


<210>  21
<211>  1663
<212>  DNA
<213>  artificial sequence

<220>
<223>  CAG promoter #2

<400>  21
ctagttatta atagtaatca attacggggt cattagttca tagcccatat atggagttcc       60

gcgttacata acttacggta aatggcccgc ctggctgacc gcccaacgac ccccgcccat      120

tgacgtcaat aatgacgtat gttcccatag taacgccaat agggactttc cattgacgtc      180

aatgggtgga gtatttacgg taaactgccc acttggcagt acatcaagtg tatcatatgc      240

caagtacgcc ccctattgac gtcaatgacg gtaaatggcc cgcctggcat tatgcccagt      300

acatgacctt atgggacttt cctacttggc agtacatcta cgtattagtc atcgctatta      360

ccatggtcga ggtgagcccc acgttctgct tcactctccc catctccccc ccctccccac      420

ccccaatttt gtatttattt attttttaat tattttgtgc agcgatgggg gcgggggggg      480

ggggggggcg cgcgccaggc ggggcggggc ggggcgaggg gcggggcggg gcgaggcgga      540

gaggtgcggc ggcagccaat cagagcggcg cgctccgaaa gtttcctttt atggcgaggc      600

ggcggcggcg gcggccctat aaaaagcgaa gcgcgcggcg ggcgggagtc gctgcgacgc      660

tgccttcgcc ccgtgccccg ctccgccgcc gcctcgcgcc gcccgccccg gctctgactg      720

accgcgttac tcccacaggt gagcgggcgg gacggccctt ctcctccggg ctgtaattag      780

cgcttggttt aatgacggct tgtttctttt ctgtggctgc gtgaaagcct tgaggggctc      840

cgggagggcc ctttgtgcgg gggggagcgg ctcggggggt gcgtgcgtgt gtgtgtgcgt      900

ggggagcgcc gcgtgcggcc cgcgctgccc ggcggctgtg agcgctgcgg gcgcggcgcg      960

gggctttgtg cgctccgcag tgtgcgcgag gggagcgcgg ccgggggcgg tgccccgcgg     1020

tgcggggggg gctgcgaggg gaacaaaggc tgcgtgcggg gtgtgtgcgt gggggggtga     1080

gcagggggtg tgggcgcggc ggtcgggctg taaccccccc ctgcaccccc ctccccgagt     1140

tgctgagcac ggcccggctt cgggtgcggg gctccgtacg gggcgtggcg cggggctcgc     1200

cgtgccgggc ggggggtggc ggcaggtggg ggtgccgggc ggggcggggc cgcctcgggc     1260

cggggagggc tcgggggagg ggcgcggcgg cccccggagc gccggcggct gtcgaggcgc     1320

ggcgagccgc agccattgcc ttttatggta atcgtgcgag agggcgcagg gacttccttt     1380

gtcccaaatc tgtgcggagc cgaaatctgg gaggcgccgc cgcaccccct ctagcgggcg     1440

cggggcgaag cggtgcggcg ccggcaggaa ggaaatgggc ggggagggcc ttcgtgcgtc     1500

gccgcgccgc cgtccccttc tccctctcca gcctcggggc tgtccgcggg gggacggctg     1560

ccttcggggg ggacggggca gggcggggtt cggcttctgg cgtgtgaccg gcggctctag     1620

agcctctgct aaccatgttc atgccttctt ctttttccta cag                       1663


<210>  22
<211>  400
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Human Ubiquitin C (UbC) promoter short version

<400>  22
ggcctccgcg ccgggttttg gcgcctcccg cgggcgcccc cctcctcacg gcgagcgctg       60

ccacgtcaga cgaagggcgc agcgagcgtc ctgatccttc cgcccggacg ctcaggacag      120

cggcccgctg ctcataagac tcggccttag aaccccagta tcagcagaag gacattttag      180

gacgggactt gggtgactct agggcactgg ttttctttcc agagagcgga acaggcgagg      240

aaaagtagtc ccttctcggc gattctgcgg agggatctcc gtggggcggt gaacgccgat      300

gattatataa ggacgcgccg ggtgtggcac agctagttcc gtcgcagccg ggatttgggt      360

cgcggttctt gtttgtggat cgctgtgatc gtcacttggt                            400


<210>  23
<211>  1212
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Human Ubiquitin C (UbC) promoter long version

<400>  23
ccggaggcgc ggcccaaaac cgcggagggc gcccgcgggg ggaggagtgc cgctcgcgac       60

ggtgcagtct gcttcccgcg tcgctcgcag gactaggaag gcgggcctgc gagtcctgtc      120

gccgggcgac gagtattctg agccggaatc ttggggtcat agtcgtcttc ctgtaaaatc      180

ctgccctgaa cccactgaga tcccgtgacc aaaagaaagg tctctcgcct tgtccgctcc      240

ttttcatcag ggaagagccg ctaagacgcc tccctagagg caccccgcca cttgcggcta      300

ctaatatatt cctgcgcggc ccacaccgtg tcgatcaagg cagcgtcggc cctaaaccca      360

gcgccaagaa caaacaccta gcgacactag cagtgaacca ctcatcgccc gacgacccga      420

ccggccccga aagcaccggc ggcccggcga gccaccctgc cttcgcacac ctctctggcg      480

gttcccgaca tcagacccag gcgctcgttc caacgggact tgacccccaa cccccctcgc      540

gtcgttttac cgccgacaag ggctcagaac ttaccttctg cgaacactcc gcccgacact      600

ccagcaactt tgttccaccc cccgtaccac ccgccgttct tgggttccag aactccggaa      660

gcgattacgc cctttcgaga ataagcccac tctacccgac cccgtggtag acccctggga      720

ctgcacttca aacagtgact gacctcttga gccaaacagc agacaacgcc cccgccgtca      780

ataccgccac ggcaacccgt cacgtgggca tggaaaccct cgcgcgcggg agcagcacag      840

cactgcagtg ggcaagacaa ccgaatatta cgtcccaccc cggtggacgg ccatccacac      900

gccatccgaa aagaggcagc gtcctgcgtc ccaagcccgg atcccatccg agaggactta      960

gctgtccgcg gcctggagac cactcccctc cctattcact ccgcagtcaa agaaaccagc     1020

caaaatacat ggatagaaga attcatcgac ttcgaggcca aaacttgata cgcgagcccc     1080

aaccgctcac acaaaacact tcaaaaaatc cgtggaaaac tttacattag taaacccagt     1140

tatacattaa aagtcacaat ctgatcattt aacaggcgat ttaagaccgg caaaaaccga     1200

aaaaacaatc tg                                                         1212


<210>  24
<211>  511
<212>  DNA
<213>  Artificial sequence

<220>
<223>  Phosphoglycerate kinase 1 (PGK) promoter

<400>  24
gacccctctc tccagccact aagccagttg ctccctcggc tgacggctgc acgcgaggcc       60

tccgaacgtc ttacgccttg tggcgcgccc gtccttgtcc cgggtgtgat ggcggggtgt      120

ggggcggagg gcgtggcggg gaagggccgg cgacgagagc cgcgcgggac gactcgtcgg      180

cgataaccgg tgtcgggtag cgccagccgc gcgacggtaa cgagggaccg cgacaggcag      240

acgctcccat gatcactctg cacgccgaag gcaaacagtg caggccgtgc ggcgcttggc      300

gttccttgga agggctgaat ccccgcctcg tccttcgcag cggccccccg ggtgttccca      360

tcgccgcttc taggcccact gcgacgcttg cctgcacttc ttacacgctc tgggtcccag      420

ccgcggcgac gcaaagggcc ttggtgcggg tctcgtcggc gcagggacgc gtttgggtcc      480

cgacggaacc ttttccgcgt tggggttggg g                                     511


<210>  25
<211>  741
<212>  DNA
<213>  Artificial sequence

<220>
<223>  CBA/CBh promoter

<400>  25
agatgtactg ccaagtagga aagtcccgta aggtcatgta ctgggcataa tgccaggcgg       60

gccatttacc gtcattgacg tcaatagggg gcgtacttgg catatgatac acttgatgta      120

ctgccaagtg ggcagtttac cgtaaatact ccacccattg acgtcaatgg aaagtcccta      180

ttggcgttac tatgggaaca tacgtcatta ttgacgtcaa tgggcggggg tcgttgggcg      240

gtcagccagg cgggccattt accgtaagtt atgtaacgcg gaactccata tatgggctat      300

gaactaatga ccccgtaatt gattactatt aaccacgttc tgcttcactc tccccatctc      360

ccccccctcc ccacccccaa ttttgtattt atttattttt taattatttt gtgcagcgat      420

gggggcgggg gggggggggg cgcgcgccag gcggggcggg gcggggcgag gggcggggcg      480

gggcgaggcg gagaggtgcg gcggcagcca atcagagcgg cgcgctccga aagtttcctt      540

ttatggcgag gcggcggcgg cggcggccct ataaaaagcg aagcgcgcgg cggaactgaa      600

aaaccagaaa gttaactggt aagtttagtc tttttgtctt ttatttcagg tcctggtggt      660

gcaaatcaaa gaactgctcc tcagtggatg ttgcctttac ttctaggcct gtacggaagt      720

gttacttctg ctctaaaagc t                                                741


<210>  26
<211>  818
<212>  DNA
<213>  Artificial sequence

<220>
<223>  CBA/CBh promoter

<400>  26
agatgtactg ccaagtagga aagtcccgta aggtcatgta ctgggcataa tgccaggcgg       60

gccatttacc gtcattgacg tcaatagggg gcgtacttgg catatgatac acttgatgta      120

ctgccaagtg ggcagtttac cgtaaatact ccacccattg acgtcaatgg aaagtcccta      180

ttggcgttac tatgggaaca tacgtcatta ttgacgtcaa tgggcggggg tcgttgggcg      240

gtcagccagg cgggccattt accgtaagtt atgtaacgcg gaactccata tatgggctat      300

gaactaatga ccccgtaatt gattactatt aaccacgttc tgcttcactc tccccatctc      360

ccccccctcc ccacccccaa ttttgtattt atttattttt taattatttt gtgcagcgat      420

gggggcgggg gggggggggg cgcgcgccag gcggggcggg gcggggcgag gggcggggcg      480

gggcgaggcg gagaggtgcg gcggcagcca atcagagcgg cgcgctccga aagtttcctt      540

ttatggcgag gcggcggcgg cggcggccct ataaaaagcg aagcgcgcgg cggggagtcg      600

ctgcgttgcc ttcgccccgt gccccgctcc gcgccgcctc gcgccgcccg ccccggctct      660

gactgaccgc gttactccca caggtgagcg ggcgggacgg cccttctcct ccgggctgta      720

attagcaaga ggtaagggtt taagggatgg ttggttggtg gggtattaat gtttaattac      780

ctgttttaca ggcctgaaat cacttggttt taggttgg                              818


<210>  27
<211>  195
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide sequence of the JeT promoter

<400>  27
gaattcgggc ggagttaggg cggagccaat cagcgtgcgc cgttccgaaa gttgcctttt       60

atggctgggc ggagaatggg cggtgaacgc cgatgattat ataaggacgc gccgggtgtg      120

gcacagctag ttccgtcgca gccgggattt gggtcgcggt tcttgtttgt ggatccctgt      180

gatcgtcact tgaca                                                       195


<210>  28
<211>  510
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Nucleotide sequence of the Human Growth Hormone polyA

<400>  28
acgggtggca tccctgtgac ccctccccag tgcctctcct ggccctggaa gttgccactc       60

cagtgcccac cagccttgtc ctaataaaat taagttgcat cattttgtct gactaggtgt      120

ccttctataa tattatgggg tggagggggg tggtatggag caaggggcaa gttgggaaga      180

caacctgtag ggcctgcggg gtctattggg aaccaagctg gagtgcagtg gcacaatctt      240

ggctcactgc aatctccgcc tcctgggttc aagcgattct cctgcctcag cctcccgagt      300

tgttgggatt ccaggcatgc atgaccaggc tcagctaatt tttgtttttt tggtagagac      360

ggggtttcac catattggcc aggctggtct ccaactccta atctcaggtg atctacccac      420

cttggcctcc caaattgctg ggattacagg cgtgaaccac tgctcccttc cctgtccttc      480

tgattttgta ggtaaccacg tgcggaccga                                       510


