                         SEQUENCE LISTING

<110>  UCL BUSINESS PLC.
 
<120>  GENE THERAPY

<130>  N414218WO-A

<150>  GB 1811540.2
<151>  2018-07-13

<150>  PCT/GB2019/050214
<151>  2019-01-25

<150>  GB 1811541.0
<151>  2018-07-13

<160>  21    

<170>  PatentIn version 3.5

<210>  1
<211>  1611
<212>  DNA
<213>  Homo sapiens

<400>  1
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>  2
<211>  260
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CBA promoter

<400>  2
ccacgttctg cttcactctc cccatctccc ccccctcccc acccccaatt ttgtatttat       60

ttatttttta attattttgt gcagcgatgg gggcgggggg ggggggggcg cgcgccaggc      120

ggggcggggc ggggcgaggg gcggggcggg gcgaggcgga gaggtgcggc ggcagccaat      180

cagagcggcg cgctccgaaa gtttcctttt atggcgaggc ggcggcggcg gcggccctat      240

aaaaagcgaa gcgcgcggcg                                                  260


<210>  3
<211>  643
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CAG promoter

<400>  3
tagttattaa tagtaatcaa ttacggggtc attagttcat agcccatata tggagttccg       60

cgttacataa cttacggtaa atggcccgcc tggctgaccg cccaacgacc cccgcccatt      120

gacgtcaata atgacgtatg ttcccatagt aacgccaata gggactttcc attgacgtca      180

atgggtggag tatttacggt aaactgccca cttggcagta catcaagtgt atcatatgcc      240

aagtacgccc cctattgacg tcaatgacgg taaatggccc gcctggcatt atgcccagta      300

catgacctta tgggactttc ctacttggca gtacatctac gtattagtca tcgctattac      360

catggtcgag gtgagcccca cgttctgctt cactctcccc atctcccccc cctccccacc      420

cccaattttg tatttattta ttttttaatt attttgtgca gcgatggggg cggggggggg      480

gggggggcgc gcgccaggcg gggcggggcg gggcgagggg cggggcgggg cgaggcggag      540

aggtgcggcg gcagccaatc agagcggcgc gctccgaaag tttcctttta tggcgaggcg      600

gcggcggcgg cggccctata aaaagcgaag cgcgcggcgg gcg                        643


<210>  4
<211>  609
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  WPRE element

<400>  4
gcttatcgat aatcaacctc tggattacaa aatttgtgaa agattgactg gtattcttaa       60

ctatgttgct ccttttacgc tatgtggata cgctgcttta atgcctttgt atcatgctat      120

tgcttcccgt atggctttca ttttctcctc cttgtataaa tcctggttgc tgtctcttta      180

tgaggagttg tggcccgttg tcaggcaacg tggcgtggtg tgcactgtgt ttgctgacgc      240

aacccccact ggttggggca ttgccaccac ctgtcagctc ctttccggga ctttcgcttt      300

ccccctccct attgccacgg cggaactcat cgccgcctgc cttgcccgct gctggacagg      360

ggctcggctg ttgggcactg acaattccgt ggtgttgtcg gggaaatcat cgtcctttcc      420

ttggctgctc gcctatgttg ccacctggat tctgcgcggg acgtccttct gctacgtccc      480

ttcggccctc aatccagcgg accttccttc ccgcggcctg ctgccggctc tgcggcctct      540

tccgcgtctt cgccttcgcc ctcagacgag tcggatctcc ctttgggccg cctccccgca      600

tcgataccg                                                              609


<210>  5
<211>  1908
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CBA GBA1 expression construct

<400>  5
ccacgttctg cttcactctc cccatctccc ccccctcccc acccccaatt ttgtatttat       60

ttatttttta attattttgt gcagcgatgg gggcgggggg ggggggggcg cgcgccaggc      120

ggggcggggc ggggcgaggg gcggggcggg gcgaggcgga gaggtgcggc ggcagccaat      180

cagagcggcg cgctccgaaa gtttcctttt atggcgaggc ggcggcggcg gcggccctat      240

aaaaagcgaa gcgcgcggcg gggtaccgga tcctctagag tcgactccgg agccaccatg      300

gagttttcaa gtccttccag agaggaatgt cccaagcctt tgagtagggt aagcatcatg      360

gctggcagcc tcacaggatt gcttctactt caggcagtgt cgtgggcatc aggtgcccgc      420

ccctgcatcc ctaaaagctt cggctacagc tcggtggtgt gtgtctgcaa tgccacatac      480

tgtgactcct ttgacccccc gacctttcct gcccttggta ccttcagccg ctatgagagt      540

acacgcagtg ggcgacggat ggagctgagt atggggccca tccaggctaa tcacacgggc      600

acaggcctgc tactgaccct gcagccagaa cagaagttcc agaaagtgaa gggatttgga      660

ggggccatga cagatgctgc tgctctcaac atccttgccc tgtcaccccc tgcccaaaat      720

ttgctactta aatcgtactt ctctgaagaa ggaatcggat ataacatcat ccgggtaccc      780

atggccagct gtgacttctc catccgcacc tacacctatg cagacacccc tgatgatttc      840

cagttgcaca acttcagcct cccagaggaa gataccaagc tcaagatacc cctgattcac      900

cgagccctgc agttggccca gcgtcccgtt tcactccttg ccagcccctg gacatcaccc      960

acttggctca agaccaatgg agcggtgaat gggaaggggt cactcaaggg acagcccgga     1020

gacatctacc accagacctg ggccagatac tttgtgaagt tcctggatgc ctatgctgag     1080

cacaagttac agttctgggc agtgacagct gaaaatgagc cttctgctgg gctgttgagt     1140

ggatacccct tccagtgcct gggcttcacc cctgaacatc agcgagactt cattgcccgt     1200

gacctaggtc ctaccctcgc caacagtact caccacaatg tccgcctact catgctggat     1260

gaccaacgct tgctgctgcc ccactgggca aaggtggtac tgacagaccc agaagcagct     1320

aaatatgttc atggcattgc tgtacattgg tacctggact ttctggctcc agccaaagcc     1380

accctagggg agacacaccg cctgttcccc aacaccatgc tctttgcctc agaggcctgt     1440

gtgggctcca agttctggga gcagagtgtg cggctaggct cctgggatcg agggatgcag     1500

tacagccaca gcatcatcac gaacctcctg taccatgtgg tcggctggac cgactggaac     1560

cttgccctga accccgaagg aggacccaat tgggtgcgta actttgtcga cagtcccatc     1620

attgtagaca tcaccaagga cacgttttac aaacagccca tgttctacca ccttggccac     1680

ttcagcaagt tcattcctga gggctcccag agagtggggc tggttgccag tcagaagaac     1740

gacctggacg cagtggcact gatgcatccc gatggctctg ctgttgtggt cgtgctaaac     1800

cgctcctcta aggatgtgcc tcttaccatc aaggatcctg ctgtgggctt cctggagaca     1860

atctcacctg gctactccat tcacacctac ctgtggcgtc gccagtga                  1908


<210>  6
<211>  2291
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CAG GBA1 expression construct

<400>  6
tagttattaa tagtaatcaa ttacggggtc attagttcat agcccatata tggagttccg       60

cgttacataa cttacggtaa atggcccgcc tggctgaccg cccaacgacc cccgcccatt      120

gacgtcaata atgacgtatg ttcccatagt aacgccaata gggactttcc attgacgtca      180

atgggtggag tatttacggt aaactgccca cttggcagta catcaagtgt atcatatgcc      240

aagtacgccc cctattgacg tcaatgacgg taaatggccc gcctggcatt atgcccagta      300

catgacctta tgggactttc ctacttggca gtacatctac gtattagtca tcgctattac      360

catggtcgag gtgagcccca cgttctgctt cactctcccc atctcccccc cctccccacc      420

cccaattttg tatttattta ttttttaatt attttgtgca gcgatggggg cggggggggg      480

gggggggcgc gcgccaggcg gggcggggcg gggcgagggg cggggcgggg cgaggcggag      540

aggtgcggcg gcagccaatc agagcggcgc gctccgaaag tttcctttta tggcgaggcg      600

gcggcggcgg cggccctata aaaagcgaag cgcgcggcgg gcggggtacc ggatcctcta      660

gagtcgactc cggagccacc atggagtttt caagtccttc cagagaggaa tgtcccaagc      720

ctttgagtag ggtaagcatc atggctggca gcctcacagg attgcttcta cttcaggcag      780

tgtcgtgggc atcaggtgcc cgcccctgca tccctaaaag cttcggctac agctcggtgg      840

tgtgtgtctg caatgccaca tactgtgact cctttgaccc cccgaccttt cctgcccttg      900

gtaccttcag ccgctatgag agtacacgca gtgggcgacg gatggagctg agtatggggc      960

ccatccaggc taatcacacg ggcacaggcc tgctactgac cctgcagcca gaacagaagt     1020

tccagaaagt gaagggattt ggaggggcca tgacagatgc tgctgctctc aacatccttg     1080

ccctgtcacc ccctgcccaa aatttgctac ttaaatcgta cttctctgaa gaaggaatcg     1140

gatataacat catccgggta cccatggcca gctgtgactt ctccatccgc acctacacct     1200

atgcagacac ccctgatgat ttccagttgc acaacttcag cctcccagag gaagatacca     1260

agctcaagat acccctgatt caccgagccc tgcagttggc ccagcgtccc gtttcactcc     1320

ttgccagccc ctggacatca cccacttggc tcaagaccaa tggagcggtg aatgggaagg     1380

ggtcactcaa gggacagccc ggagacatct accaccagac ctgggccaga tactttgtga     1440

agttcctgga tgcctatgct gagcacaagt tacagttctg ggcagtgaca gctgaaaatg     1500

agccttctgc tgggctgttg agtggatacc ccttccagtg cctgggcttc acccctgaac     1560

atcagcgaga cttcattgcc cgtgacctag gtcctaccct cgccaacagt actcaccaca     1620

atgtccgcct actcatgctg gatgaccaac gcttgctgct gccccactgg gcaaaggtgg     1680

tactgacaga cccagaagca gctaaatatg ttcatggcat tgctgtacat tggtacctgg     1740

actttctggc tccagccaaa gccaccctag gggagacaca ccgcctgttc cccaacacca     1800

tgctctttgc ctcagaggcc tgtgtgggct ccaagttctg ggagcagagt gtgcggctag     1860

gctcctggga tcgagggatg cagtacagcc acagcatcat cacgaacctc ctgtaccatg     1920

tggtcggctg gaccgactgg aaccttgccc tgaaccccga aggaggaccc aattgggtgc     1980

gtaactttgt cgacagtccc atcattgtag acatcaccaa ggacacgttt tacaaacagc     2040

ccatgttcta ccaccttggc cacttcagca agttcattcc tgagggctcc cagagagtgg     2100

ggctggttgc cagtcagaag aacgacctgg acgcagtggc actgatgcat cccgatggct     2160

ctgctgttgt ggtcgtgcta aaccgctcct ctaaggatgt gcctcttacc atcaaggatc     2220

ctgctgtggg cttcctggag acaatctcac ctggctactc cattcacacc tacctgtggc     2280

gtcgccagtg a                                                          2291


<210>  7
<211>  2531
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CBA GBA1 WPRE expression construct

<400>  7
ccacgttctg cttcactctc cccatctccc ccccctcccc acccccaatt ttgtatttat       60

ttatttttta attattttgt gcagcgatgg gggcgggggg ggggggggcg cgcgccaggc      120

ggggcggggc ggggcgaggg gcggggcggg gcgaggcgga gaggtgcggc ggcagccaat      180

cagagcggcg cgctccgaaa gtttcctttt atggcgaggc ggcggcggcg gcggccctat      240

aaaaagcgaa gcgcgcggcg gggtaccgga tcctctagag tcgactccgg agccaccatg      300

gagttttcaa gtccttccag agaggaatgt cccaagcctt tgagtagggt aagcatcatg      360

gctggcagcc tcacaggatt gcttctactt caggcagtgt cgtgggcatc aggtgcccgc      420

ccctgcatcc ctaaaagctt cggctacagc tcggtggtgt gtgtctgcaa tgccacatac      480

tgtgactcct ttgacccccc gacctttcct gcccttggta ccttcagccg ctatgagagt      540

acacgcagtg ggcgacggat ggagctgagt atggggccca tccaggctaa tcacacgggc      600

acaggcctgc tactgaccct gcagccagaa cagaagttcc agaaagtgaa gggatttgga      660

ggggccatga cagatgctgc tgctctcaac atccttgccc tgtcaccccc tgcccaaaat      720

ttgctactta aatcgtactt ctctgaagaa ggaatcggat ataacatcat ccgggtaccc      780

atggccagct gtgacttctc catccgcacc tacacctatg cagacacccc tgatgatttc      840

cagttgcaca acttcagcct cccagaggaa gataccaagc tcaagatacc cctgattcac      900

cgagccctgc agttggccca gcgtcccgtt tcactccttg ccagcccctg gacatcaccc      960

acttggctca agaccaatgg agcggtgaat gggaaggggt cactcaaggg acagcccgga     1020

gacatctacc accagacctg ggccagatac tttgtgaagt tcctggatgc ctatgctgag     1080

cacaagttac agttctgggc agtgacagct gaaaatgagc cttctgctgg gctgttgagt     1140

ggatacccct tccagtgcct gggcttcacc cctgaacatc agcgagactt cattgcccgt     1200

gacctaggtc ctaccctcgc caacagtact caccacaatg tccgcctact catgctggat     1260

gaccaacgct tgctgctgcc ccactgggca aaggtggtac tgacagaccc agaagcagct     1320

aaatatgttc atggcattgc tgtacattgg tacctggact ttctggctcc agccaaagcc     1380

accctagggg agacacaccg cctgttcccc aacaccatgc tctttgcctc agaggcctgt     1440

gtgggctcca agttctggga gcagagtgtg cggctaggct cctgggatcg agggatgcag     1500

tacagccaca gcatcatcac gaacctcctg taccatgtgg tcggctggac cgactggaac     1560

cttgccctga accccgaagg aggacccaat tgggtgcgta actttgtcga cagtcccatc     1620

attgtagaca tcaccaagga cacgttttac aaacagccca tgttctacca ccttggccac     1680

ttcagcaagt tcattcctga gggctcccag agagtggggc tggttgccag tcagaagaac     1740

gacctggacg cagtggcact gatgcatccc gatggctctg ctgttgtggt cgtgctaaac     1800

cgctcctcta aggatgtgcc tcttaccatc aaggatcctg ctgtgggctt cctggagaca     1860

atctcacctg gctactccat tcacacctac ctgtggcgtc gccagtgaga attcgatatc     1920

aagcttatcg ataatcaacc tctggattac aaaatttgtg aaagattgac tggtattctt     1980

aactatgttg ctccttttac gctatgtgga tacgctgctt taatgccttt gtatcatgct     2040

attgcttccc gtatggcttt cattttctcc tccttgtata aatcctggtt gctgtctctt     2100

tatgaggagt tgtggcccgt tgtcaggcaa cgtggcgtgg tgtgcactgt gtttgctgac     2160

gcaaccccca ctggttgggg cattgccacc acctgtcagc tcctttccgg gactttcgct     2220

ttccccctcc ctattgccac ggcggaactc atcgccgcct gccttgcccg ctgctggaca     2280

ggggctcggc tgttgggcac tgacaattcc gtggtgttgt cggggaaatc atcgtccttt     2340

ccttggctgc tcgcctatgt tgccacctgg attctgcgcg ggacgtcctt ctgctacgtc     2400

ccttcggccc tcaatccagc ggaccttcct tcccgcggcc tgctgccggc tctgcggcct     2460

cttccgcgtc ttcgccttcg ccctcagacg agtcggatct ccctttgggc cgcctccccg     2520

catcgatacc g                                                          2531


<210>  8
<211>  2914
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CAG GBA1 WPRE expression construct

<400>  8
tagttattaa tagtaatcaa ttacggggtc attagttcat agcccatata tggagttccg       60

cgttacataa cttacggtaa atggcccgcc tggctgaccg cccaacgacc cccgcccatt      120

gacgtcaata atgacgtatg ttcccatagt aacgccaata gggactttcc attgacgtca      180

atgggtggag tatttacggt aaactgccca cttggcagta catcaagtgt atcatatgcc      240

aagtacgccc cctattgacg tcaatgacgg taaatggccc gcctggcatt atgcccagta      300

catgacctta tgggactttc ctacttggca gtacatctac gtattagtca tcgctattac      360

catggtcgag gtgagcccca cgttctgctt cactctcccc atctcccccc cctccccacc      420

cccaattttg tatttattta ttttttaatt attttgtgca gcgatggggg cggggggggg      480

gggggggcgc gcgccaggcg gggcggggcg gggcgagggg cggggcgggg cgaggcggag      540

aggtgcggcg gcagccaatc agagcggcgc gctccgaaag tttcctttta tggcgaggcg      600

gcggcggcgg cggccctata aaaagcgaag cgcgcggcgg gcggggtacc ggatcctcta      660

gagtcgactc cggagccacc atggagtttt caagtccttc cagagaggaa tgtcccaagc      720

ctttgagtag ggtaagcatc atggctggca gcctcacagg attgcttcta cttcaggcag      780

tgtcgtgggc atcaggtgcc cgcccctgca tccctaaaag cttcggctac agctcggtgg      840

tgtgtgtctg caatgccaca tactgtgact cctttgaccc cccgaccttt cctgcccttg      900

gtaccttcag ccgctatgag agtacacgca gtgggcgacg gatggagctg agtatggggc      960

ccatccaggc taatcacacg ggcacaggcc tgctactgac cctgcagcca gaacagaagt     1020

tccagaaagt gaagggattt ggaggggcca tgacagatgc tgctgctctc aacatccttg     1080

ccctgtcacc ccctgcccaa aatttgctac ttaaatcgta cttctctgaa gaaggaatcg     1140

gatataacat catccgggta cccatggcca gctgtgactt ctccatccgc acctacacct     1200

atgcagacac ccctgatgat ttccagttgc acaacttcag cctcccagag gaagatacca     1260

agctcaagat acccctgatt caccgagccc tgcagttggc ccagcgtccc gtttcactcc     1320

ttgccagccc ctggacatca cccacttggc tcaagaccaa tggagcggtg aatgggaagg     1380

ggtcactcaa gggacagccc ggagacatct accaccagac ctgggccaga tactttgtga     1440

agttcctgga tgcctatgct gagcacaagt tacagttctg ggcagtgaca gctgaaaatg     1500

agccttctgc tgggctgttg agtggatacc ccttccagtg cctgggcttc acccctgaac     1560

atcagcgaga cttcattgcc cgtgacctag gtcctaccct cgccaacagt actcaccaca     1620

atgtccgcct actcatgctg gatgaccaac gcttgctgct gccccactgg gcaaaggtgg     1680

tactgacaga cccagaagca gctaaatatg ttcatggcat tgctgtacat tggtacctgg     1740

actttctggc tccagccaaa gccaccctag gggagacaca ccgcctgttc cccaacacca     1800

tgctctttgc ctcagaggcc tgtgtgggct ccaagttctg ggagcagagt gtgcggctag     1860

gctcctggga tcgagggatg cagtacagcc acagcatcat cacgaacctc ctgtaccatg     1920

tggtcggctg gaccgactgg aaccttgccc tgaaccccga aggaggaccc aattgggtgc     1980

gtaactttgt cgacagtccc atcattgtag acatcaccaa ggacacgttt tacaaacagc     2040

ccatgttcta ccaccttggc cacttcagca agttcattcc tgagggctcc cagagagtgg     2100

ggctggttgc cagtcagaag aacgacctgg acgcagtggc actgatgcat cccgatggct     2160

ctgctgttgt ggtcgtgcta aaccgctcct ctaaggatgt gcctcttacc atcaaggatc     2220

ctgctgtggg cttcctggag acaatctcac ctggctactc cattcacacc tacctgtggc     2280

gtcgccagtg agaattcgat atcaagctta tcgataatca acctctggat tacaaaattt     2340

gtgaaagatt gactggtatt cttaactatg ttgctccttt tacgctatgt ggatacgctg     2400

ctttaatgcc tttgtatcat gctattgctt cccgtatggc tttcattttc tcctccttgt     2460

ataaatcctg gttgctgtct ctttatgagg agttgtggcc cgttgtcagg caacgtggcg     2520

tggtgtgcac tgtgtttgct gacgcaaccc ccactggttg gggcattgcc accacctgtc     2580

agctcctttc cgggactttc gctttccccc tccctattgc cacggcggaa ctcatcgccg     2640

cctgccttgc ccgctgctgg acaggggctc ggctgttggg cactgacaat tccgtggtgt     2700

tgtcggggaa atcatcgtcc tttccttggc tgctcgccta tgttgccacc tggattctgc     2760

gcgggacgtc cttctgctac gtcccttcgg ccctcaatcc agcggacctt ccttcccgcg     2820

gcctgctgcc ggctctgcgg cctcttccgc gtcttcgcct tcgccctcag acgagtcgga     2880

tctccctttg ggccgcctcc ccgcatcgat accg                                 2914


<210>  9
<211>  5961
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  pAAV.CBA.hGBA1.WPRE.hGHpA

<400>  9
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 agaattcgat     1620

atcaagctta tcgataatca acctctggat tacaaaattt gtgaaagatt gactggtatt     1680

cttaactatg ttgctccttt tacgctatgt ggatacgctg ctttaatgcc tttgtatcat     1740

gctattgctt cccgtatggc tttcattttc tcctccttgt ataaatcctg gttgctgtct     1800

ctttatgagg agttgtggcc cgttgtcagg caacgtggcg tggtgtgcac tgtgtttgct     1860

gacgcaaccc ccactggttg gggcattgcc accacctgtc agctcctttc cgggactttc     1920

gctttccccc tccctattgc cacggcggaa ctcatcgccg cctgccttgc ccgctgctgg     1980

acaggggctc ggctgttggg cactgacaat tccgtggtgt tgtcggggaa atcatcgtcc     2040

tttccttggc tgctcgccta tgttgccacc tggattctgc gcgggacgtc cttctgctac     2100

gtcccttcgg ccctcaatcc agcggacctt ccttcccgcg gcctgctgcc ggctctgcgg     2160

cctcttccgc gtcttcgcct tcgccctcag acgagtcgga tctccctttg ggccgcctcc     2220

ccgcatcgat accgagcgct gctcgagaga tctacgggtg gcatccctgt gacccctccc     2280

cagtgcctct cctggccctg gaagttgcca ctccagtgcc caccagcctt gtcctaataa     2340

aattaagttg catcattttg tctgactagg tgtccttcta taatattatg gggtggaggg     2400

gggtggtatg gagcaagggg caagttggga agacaacctg tagggcctgc ggggtctatt     2460

gggaaccaag ctggagtgca gtggcacaat cttggctcac tgcaatctcc gcctcctggg     2520

ttcaagcgat tctcctgcct cagcctcccg agttgttggg attccaggca tgcatgacca     2580

ggctcagcta atttttgttt ttttggtaga gacggggttt caccatattg gccaggctgg     2640

tctccaactc ctaatctcag gtgatctacc caccttggcc tcccaaattg ctgggattac     2700

aggcgtgaac cactgctccc ttccctgtcc ttctgatttt gtaggtaacc acgtgcggac     2760

cgagcggccg caggaacccc tagtgatgga gttggccact ccctctctgc gcgctcgctc     2820

gctcactgag gccgggcgac caaaggtcgc ccgacgcccg ggctttgccc gggcggcctc     2880

agtgagcgag cgagcgcgca gctgcctgca ggggcgcctg atgcggtatt ttctccttac     2940

gcatctgtgc ggtatttcac accgcatacg tcaaagcaac catagtacgc gccctgtagc     3000

ggcgcattaa gcgcggcggg tgtggtggtt acgcgcagcg tgaccgctac acttgccagc     3060

gccctagcgc ccgctccttt cgctttcttc ccttcctttc tcgccacgtt cgccggcttt     3120

ccccgtcaag ctctaaatcg ggggctccct ttagggttcc gatttagtgc tttacggcac     3180

ctcgacccca aaaaacttga tttgggtgat ggttcacgta gtgggccatc gccctgatag     3240

acggtttttc gccctttgac gttggagtcc acgttcttta atagtggact cttgttccaa     3300

actggaacaa cactcaaccc tatctcgggc tattcttttg atttataagg gattttgccg     3360

atttcggcct attggttaaa aaatgagctg atttaacaaa aatttaacgc gaattttaac     3420

aaaatattaa cgtttacaat tttatggtgc actctcagta caatctgctc tgatgccgca     3480

tagttaagcc agccccgaca cccgccaaca cccgctgacg cgccctgacg ggcttgtctg     3540

ctcccggcat ccgcttacag acaagctgtg accgtctccg ggagctgcat gtgtcagagg     3600

ttttcaccgt catcaccgaa acgcgcgaga cgaaagggcc tcgtgatacg cctattttta     3660

taggttaatg tcatgataat aatggtttct tagacgtcag gtggcacttt tcggggaaat     3720

gtgcgcggaa cccctatttg tttatttttc taaatacatt caaatatgta tccgctcatg     3780

agacaataac cctgataaat gcttcaataa tattgaaaaa ggaagagtat gagtattcaa     3840

catttccgtg tcgcccttat tccctttttt gcggcatttt gccttcctgt ttttgctcac     3900

ccagaaacgc tggtgaaagt aaaagatgct gaagatcagt tgggtgcacg agtgggttac     3960

atcgaactgg atctcaacag cggtaagatc cttgagagtt ttcgccccga agaacgtttt     4020

ccaatgatga gcacttttaa agttctgcta tgtggcgcgg tattatcccg tattgacgcc     4080

gggcaagagc aactcggtcg ccgcatacac tattctcaga atgacttggt tgagtactca     4140

ccagtcacag aaaagcatct tacggatggc atgacagtaa gagaattatg cagtgctgcc     4200

ataaccatga gtgataacac tgcggccaac ttacttctga caacgatcgg aggaccgaag     4260

gagctaaccg cttttttgca caacatgggg gatcatgtaa ctcgccttga tcgttgggaa     4320

ccggagctga atgaagccat accaaacgac gagcgtgaca ccacgatgcc tgtagcaatg     4380

gcaacaacgt tgcgcaaact attaactggc gaactactta ctctagcttc ccggcaacaa     4440

ttaatagact ggatggaggc ggataaagtt gcaggaccac ttctgcgctc ggcccttccg     4500

gctggctggt ttattgctga taaatctgga gccggtgagc gtgggtctcg cggtatcatt     4560

gcagcactgg ggccagatgg taagccctcc cgtatcgtag ttatctacac gacggggagt     4620

caggcaacta tggatgaacg aaatagacag atcgctgaga taggtgcctc actgattaag     4680

cattggtaac tgtcagacca agtttactca tatatacttt agattgattt aaaacttcat     4740

ttttaattta aaaggatcta ggtgaagatc ctttttgata atctcatgac caaaatccct     4800

taacgtgagt tttcgttcca ctgagcgtca gaccccgtag aaaagatcaa aggatcttct     4860

tgagatcctt tttttctgcg cgtaatctgc tgcttgcaaa caaaaaaacc accgctacca     4920

gcggtggttt gtttgccgga tcaagagcta ccaactcttt ttccgaaggt aactggcttc     4980

agcagagcgc agataccaaa tactgtcctt ctagtgtagc cgtagttagg ccaccacttc     5040

aagaactctg tagcaccgcc tacatacctc gctctgctaa tcctgttacc agtggctgct     5100

gccagtggcg ataagtcgtg tcttaccggg ttggactcaa gacgatagtt accggataag     5160

gcgcagcggt cgggctgaac ggggggttcg tgcacacagc ccagcttgga gcgaacgacc     5220

tacaccgaac tgagatacct acagcgtgag ctatgagaaa gcgccacgct tcccgaaggg     5280

agaaaggcgg acaggtatcc ggtaagcggc agggtcggaa caggagagcg cacgagggag     5340

cttccagggg gaaacgcctg gtatctttat agtcctgtcg ggtttcgcca cctctgactt     5400

gagcgtcgat ttttgtgatg ctcgtcaggg gggcggagcc tatggaaaaa cgccagcaac     5460

gcggcctttt tacggttcct ggccttttgc tggccttttg ctcacatgtc ctgcaggcag     5520

ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt     5580

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact     5640

aggggttcct gcggccgcac gcgtccacgt tctgcttcac tctccccatc tcccccccct     5700

ccccaccccc aattttgtat ttatttattt tttaattatt ttgtgcagcg atgggggcgg     5760

gggggggggg ggcgcgcgcc aggcggggcg gggcggggcg aggggcgggg cggggcgagg     5820

cggagaggtg cggcggcagc caatcagagc ggcgcgctcc gaaagtttcc ttttatggcg     5880

aggcggcggc ggcggcggcc ctataaaaag cgaagcgcgc ggcggggtac cggatcctct     5940

agagtcgact ccggagccac c                                               5961


<210>  10
<211>  6344
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  pAAV.CAG.hGBA1.WPRE.hGHpA

<400>  10
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 agaattcgat     1620

atcaagctta tcgataatca acctctggat tacaaaattt gtgaaagatt gactggtatt     1680

cttaactatg ttgctccttt tacgctatgt ggatacgctg ctttaatgcc tttgtatcat     1740

gctattgctt cccgtatggc tttcattttc tcctccttgt ataaatcctg gttgctgtct     1800

ctttatgagg agttgtggcc cgttgtcagg caacgtggcg tggtgtgcac tgtgtttgct     1860

gacgcaaccc ccactggttg gggcattgcc accacctgtc agctcctttc cgggactttc     1920

gctttccccc tccctattgc cacggcggaa ctcatcgccg cctgccttgc ccgctgctgg     1980

acaggggctc ggctgttggg cactgacaat tccgtggtgt tgtcggggaa atcatcgtcc     2040

tttccttggc tgctcgccta tgttgccacc tggattctgc gcgggacgtc cttctgctac     2100

gtcccttcgg ccctcaatcc agcggacctt ccttcccgcg gcctgctgcc ggctctgcgg     2160

cctcttccgc gtcttcgcct tcgccctcag acgagtcgga tctccctttg ggccgcctcc     2220

ccgcatcgat accgagcgct gctcgagaga tctacgggtg gcatccctgt gacccctccc     2280

cagtgcctct cctggccctg gaagttgcca ctccagtgcc caccagcctt gtcctaataa     2340

aattaagttg catcattttg tctgactagg tgtccttcta taatattatg gggtggaggg     2400

gggtggtatg gagcaagggg caagttggga agacaacctg tagggcctgc ggggtctatt     2460

gggaaccaag ctggagtgca gtggcacaat cttggctcac tgcaatctcc gcctcctggg     2520

ttcaagcgat tctcctgcct cagcctcccg agttgttggg attccaggca tgcatgacca     2580

ggctcagcta atttttgttt ttttggtaga gacggggttt caccatattg gccaggctgg     2640

tctccaactc ctaatctcag gtgatctacc caccttggcc tcccaaattg ctgggattac     2700

aggcgtgaac cactgctccc ttccctgtcc ttctgatttt gtaggtaacc acgtgcggac     2760

cgagcggccg caggaacccc tagtgatgga gttggccact ccctctctgc gcgctcgctc     2820

gctcactgag gccgggcgac caaaggtcgc ccgacgcccg ggctttgccc gggcggcctc     2880

agtgagcgag cgagcgcgca gctgcctgca ggggcgcctg atgcggtatt ttctccttac     2940

gcatctgtgc ggtatttcac accgcatacg tcaaagcaac catagtacgc gccctgtagc     3000

ggcgcattaa gcgcggcggg tgtggtggtt acgcgcagcg tgaccgctac acttgccagc     3060

gccctagcgc ccgctccttt cgctttcttc ccttcctttc tcgccacgtt cgccggcttt     3120

ccccgtcaag ctctaaatcg ggggctccct ttagggttcc gatttagtgc tttacggcac     3180

ctcgacccca aaaaacttga tttgggtgat ggttcacgta gtgggccatc gccctgatag     3240

acggtttttc gccctttgac gttggagtcc acgttcttta atagtggact cttgttccaa     3300

actggaacaa cactcaaccc tatctcgggc tattcttttg atttataagg gattttgccg     3360

atttcggcct attggttaaa aaatgagctg atttaacaaa aatttaacgc gaattttaac     3420

aaaatattaa cgtttacaat tttatggtgc actctcagta caatctgctc tgatgccgca     3480

tagttaagcc agccccgaca cccgccaaca cccgctgacg cgccctgacg ggcttgtctg     3540

ctcccggcat ccgcttacag acaagctgtg accgtctccg ggagctgcat gtgtcagagg     3600

ttttcaccgt catcaccgaa acgcgcgaga cgaaagggcc tcgtgatacg cctattttta     3660

taggttaatg tcatgataat aatggtttct tagacgtcag gtggcacttt tcggggaaat     3720

gtgcgcggaa cccctatttg tttatttttc taaatacatt caaatatgta tccgctcatg     3780

agacaataac cctgataaat gcttcaataa tattgaaaaa ggaagagtat gagtattcaa     3840

catttccgtg tcgcccttat tccctttttt gcggcatttt gccttcctgt ttttgctcac     3900

ccagaaacgc tggtgaaagt aaaagatgct gaagatcagt tgggtgcacg agtgggttac     3960

atcgaactgg atctcaacag cggtaagatc cttgagagtt ttcgccccga agaacgtttt     4020

ccaatgatga gcacttttaa agttctgcta tgtggcgcgg tattatcccg tattgacgcc     4080

gggcaagagc aactcggtcg ccgcatacac tattctcaga atgacttggt tgagtactca     4140

ccagtcacag aaaagcatct tacggatggc atgacagtaa gagaattatg cagtgctgcc     4200

ataaccatga gtgataacac tgcggccaac ttacttctga caacgatcgg aggaccgaag     4260

gagctaaccg cttttttgca caacatgggg gatcatgtaa ctcgccttga tcgttgggaa     4320

ccggagctga atgaagccat accaaacgac gagcgtgaca ccacgatgcc tgtagcaatg     4380

gcaacaacgt tgcgcaaact attaactggc gaactactta ctctagcttc ccggcaacaa     4440

ttaatagact ggatggaggc ggataaagtt gcaggaccac ttctgcgctc ggcccttccg     4500

gctggctggt ttattgctga taaatctgga gccggtgagc gtgggtctcg cggtatcatt     4560

gcagcactgg ggccagatgg taagccctcc cgtatcgtag ttatctacac gacggggagt     4620

caggcaacta tggatgaacg aaatagacag atcgctgaga taggtgcctc actgattaag     4680

cattggtaac tgtcagacca agtttactca tatatacttt agattgattt aaaacttcat     4740

ttttaattta aaaggatcta ggtgaagatc ctttttgata atctcatgac caaaatccct     4800

taacgtgagt tttcgttcca ctgagcgtca gaccccgtag aaaagatcaa aggatcttct     4860

tgagatcctt tttttctgcg cgtaatctgc tgcttgcaaa caaaaaaacc accgctacca     4920

gcggtggttt gtttgccgga tcaagagcta ccaactcttt ttccgaaggt aactggcttc     4980

agcagagcgc agataccaaa tactgtcctt ctagtgtagc cgtagttagg ccaccacttc     5040

aagaactctg tagcaccgcc tacatacctc gctctgctaa tcctgttacc agtggctgct     5100

gccagtggcg ataagtcgtg tcttaccggg ttggactcaa gacgatagtt accggataag     5160

gcgcagcggt cgggctgaac ggggggttcg tgcacacagc ccagcttgga gcgaacgacc     5220

tacaccgaac tgagatacct acagcgtgag ctatgagaaa gcgccacgct tcccgaaggg     5280

agaaaggcgg acaggtatcc ggtaagcggc agggtcggaa caggagagcg cacgagggag     5340

cttccagggg gaaacgcctg gtatctttat agtcctgtcg ggtttcgcca cctctgactt     5400

gagcgtcgat ttttgtgatg ctcgtcaggg gggcggagcc tatggaaaaa cgccagcaac     5460

gcggcctttt tacggttcct ggccttttgc tggccttttg ctcacatgtc ctgcaggcag     5520

ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt     5580

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact     5640

aggggttcct gcggccgcac gcgttagtta ttaatagtaa tcaattacgg ggtcattagt     5700

tcatagccca tatatggagt tccgcgttac ataacttacg gtaaatggcc cgcctggctg     5760

accgcccaac gacccccgcc cattgacgtc aataatgacg tatgttccca tagtaacgcc     5820

aatagggact ttccattgac gtcaatgggt ggagtattta cggtaaactg cccacttggc     5880

agtacatcaa gtgtatcata tgccaagtac gccccctatt gacgtcaatg acggtaaatg     5940

gcccgcctgg cattatgccc agtacatgac cttatgggac tttcctactt ggcagtacat     6000

ctacgtatta gtcatcgcta ttaccatggt cgaggtgagc cccacgttct gcttcactct     6060

ccccatctcc cccccctccc cacccccaat tttgtattta tttatttttt aattattttg     6120

tgcagcgatg ggggcggggg gggggggggg gcgcgcgcca ggcggggcgg ggcggggcga     6180

ggggcggggc ggggcgaggc ggagaggtgc ggcggcagcc aatcagagcg gcgcgctccg     6240

aaagtttcct tttatggcga ggcggcggcg gcggcggccc tataaaaagc gaagcgcgcg     6300

gcgggcgggg taccggatcc tctagagtcg actccggagc cacc                      6344


<210>  11
<211>  6186
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  pAAV.hSYN.hGBA1.WPRE.hGHpA

<400>  11
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 agaattcgat     1620

atcaagctta tcgataatca acctctggat tacaaaattt gtgaaagatt gactggtatt     1680

cttaactatg ttgctccttt tacgctatgt ggatacgctg ctttaatgcc tttgtatcat     1740

gctattgctt cccgtatggc tttcattttc tcctccttgt ataaatcctg gttgctgtct     1800

ctttatgagg agttgtggcc cgttgtcagg caacgtggcg tggtgtgcac tgtgtttgct     1860

gacgcaaccc ccactggttg gggcattgcc accacctgtc agctcctttc cgggactttc     1920

gctttccccc tccctattgc cacggcggaa ctcatcgccg cctgccttgc ccgctgctgg     1980

acaggggctc ggctgttggg cactgacaat tccgtggtgt tgtcggggaa atcatcgtcc     2040

tttccttggc tgctcgccta tgttgccacc tggattctgc gcgggacgtc cttctgctac     2100

gtcccttcgg ccctcaatcc agcggacctt ccttcccgcg gcctgctgcc ggctctgcgg     2160

cctcttccgc gtcttcgcct tcgccctcag acgagtcgga tctccctttg ggccgcctcc     2220

ccgcatcgat accgagcgct gctcgagaga tctacgggtg gcatccctgt gacccctccc     2280

cagtgcctct cctggccctg gaagttgcca ctccagtgcc caccagcctt gtcctaataa     2340

aattaagttg catcattttg tctgactagg tgtccttcta taatattatg gggtggaggg     2400

gggtggtatg gagcaagggg caagttggga agacaacctg tagggcctgc ggggtctatt     2460

gggaaccaag ctggagtgca gtggcacaat cttggctcac tgcaatctcc gcctcctggg     2520

ttcaagcgat tctcctgcct cagcctcccg agttgttggg attccaggca tgcatgacca     2580

ggctcagcta atttttgttt ttttggtaga gacggggttt caccatattg gccaggctgg     2640

tctccaactc ctaatctcag gtgatctacc caccttggcc tcccaaattg ctgggattac     2700

aggcgtgaac cactgctccc ttccctgtcc ttctgatttt gtaggtaacc acgtgcggac     2760

cgagcggccg caggaacccc tagtgatgga gttggccact ccctctctgc gcgctcgctc     2820

gctcactgag gccgggcgac caaaggtcgc ccgacgcccg ggctttgccc gggcggcctc     2880

agtgagcgag cgagcgcgca gctgcctgca ggggcgcctg atgcggtatt ttctccttac     2940

gcatctgtgc ggtatttcac accgcatacg tcaaagcaac catagtacgc gccctgtagc     3000

ggcgcattaa gcgcggcggg tgtggtggtt acgcgcagcg tgaccgctac acttgccagc     3060

gccctagcgc ccgctccttt cgctttcttc ccttcctttc tcgccacgtt cgccggcttt     3120

ccccgtcaag ctctaaatcg ggggctccct ttagggttcc gatttagtgc tttacggcac     3180

ctcgacccca aaaaacttga tttgggtgat ggttcacgta gtgggccatc gccctgatag     3240

acggtttttc gccctttgac gttggagtcc acgttcttta atagtggact cttgttccaa     3300

actggaacaa cactcaaccc tatctcgggc tattcttttg atttataagg gattttgccg     3360

atttcggcct attggttaaa aaatgagctg atttaacaaa aatttaacgc gaattttaac     3420

aaaatattaa cgtttacaat tttatggtgc actctcagta caatctgctc tgatgccgca     3480

tagttaagcc agccccgaca cccgccaaca cccgctgacg cgccctgacg ggcttgtctg     3540

ctcccggcat ccgcttacag acaagctgtg accgtctccg ggagctgcat gtgtcagagg     3600

ttttcaccgt catcaccgaa acgcgcgaga cgaaagggcc tcgtgatacg cctattttta     3660

taggttaatg tcatgataat aatggtttct tagacgtcag gtggcacttt tcggggaaat     3720

gtgcgcggaa cccctatttg tttatttttc taaatacatt caaatatgta tccgctcatg     3780

agacaataac cctgataaat gcttcaataa tattgaaaaa ggaagagtat gagtattcaa     3840

catttccgtg tcgcccttat tccctttttt gcggcatttt gccttcctgt ttttgctcac     3900

ccagaaacgc tggtgaaagt aaaagatgct gaagatcagt tgggtgcacg agtgggttac     3960

atcgaactgg atctcaacag cggtaagatc cttgagagtt ttcgccccga agaacgtttt     4020

ccaatgatga gcacttttaa agttctgcta tgtggcgcgg tattatcccg tattgacgcc     4080

gggcaagagc aactcggtcg ccgcatacac tattctcaga atgacttggt tgagtactca     4140

ccagtcacag aaaagcatct tacggatggc atgacagtaa gagaattatg cagtgctgcc     4200

ataaccatga gtgataacac tgcggccaac ttacttctga caacgatcgg aggaccgaag     4260

gagctaaccg cttttttgca caacatgggg gatcatgtaa ctcgccttga tcgttgggaa     4320

ccggagctga atgaagccat accaaacgac gagcgtgaca ccacgatgcc tgtagcaatg     4380

gcaacaacgt tgcgcaaact attaactggc gaactactta ctctagcttc ccggcaacaa     4440

ttaatagact ggatggaggc ggataaagtt gcaggaccac ttctgcgctc ggcccttccg     4500

gctggctggt ttattgctga taaatctgga gccggtgagc gtgggtctcg cggtatcatt     4560

gcagcactgg ggccagatgg taagccctcc cgtatcgtag ttatctacac gacggggagt     4620

caggcaacta tggatgaacg aaatagacag atcgctgaga taggtgcctc actgattaag     4680

cattggtaac tgtcagacca agtttactca tatatacttt agattgattt aaaacttcat     4740

ttttaattta aaaggatcta ggtgaagatc ctttttgata atctcatgac caaaatccct     4800

taacgtgagt tttcgttcca ctgagcgtca gaccccgtag aaaagatcaa aggatcttct     4860

tgagatcctt tttttctgcg cgtaatctgc tgcttgcaaa caaaaaaacc accgctacca     4920

gcggtggttt gtttgccgga tcaagagcta ccaactcttt ttccgaaggt aactggcttc     4980

agcagagcgc agataccaaa tactgtcctt ctagtgtagc cgtagttagg ccaccacttc     5040

aagaactctg tagcaccgcc tacatacctc gctctgctaa tcctgttacc agtggctgct     5100

gccagtggcg ataagtcgtg tcttaccggg ttggactcaa gacgatagtt accggataag     5160

gcgcagcggt cgggctgaac ggggggttcg tgcacacagc ccagcttgga gcgaacgacc     5220

tacaccgaac tgagatacct acagcgtgag ctatgagaaa gcgccacgct tcccgaaggg     5280

agaaaggcgg acaggtatcc ggtaagcggc agggtcggaa caggagagcg cacgagggag     5340

cttccagggg gaaacgcctg gtatctttat agtcctgtcg ggtttcgcca cctctgactt     5400

gagcgtcgat ttttgtgatg ctcgtcaggg gggcggagcc tatggaaaaa cgccagcaac     5460

gcggcctttt tacggttcct ggccttttgc tggccttttg ctcacatgtc ctgcaggcag     5520

ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt     5580

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact     5640

aggggttcct gcggccgcac gcgtgtgtct agactgcaga gggccctgcg tatgagtgca     5700

agtgggtttt aggaccagga tgaggcgggg tgggggtgcc tacctgacga ccgaccccga     5760

cccactggac aagcacccaa cccccattcc ccaaattgcg catcccctat cagagagggg     5820

gaggggaaac aggatgcggc gaggcgcgtg cgcactgcca gcttcagcac cgcggacagt     5880

gccttcgccc ccgcctggcg gcgcgcgcca ccgccgcctc agcactgaag gcgcgctgac     5940

gtcactcgcc ggtcccccgc aaactcccct tcccggccac cttggtcgcg tccgcgccgc     6000

cgccggccca gccggaccgc accacgcgag gcgcgagata ggggggcacg ggcgcgacca     6060

tctgcgctgc ggcgccggcg actcagcgct gcctcagtct gcggtgggca gcggaggagt     6120

cgtgtcgtgc ctgagagcgc agtcgagaag ggtaccggat cctctagagt cgactccgga     6180

gccacc                                                                6186


<210>  12
<211>  536
<212>  PRT
<213>  Homo sapiens

<400>  12

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>  13
<211>  1611
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Codon optimised sequence encoding GBA1

<400>  13
atggagttca gctccccttc tagggaggag tgccctaagc cactgtctcg cgtgagcatc       60

atggcaggaa gcctgaccgg actgctgctg ctgcaggccg tgtcctgggc atctggagca      120

aggccctgca tccctaagtc cttcggctac tctagcgtgg tgtgcgtgtg caacgccacc      180

tattgtgact ccttcgatcc ccctaccttt cccgccctgg gcacattttc tcggtacgag      240

agcacaagat ccggccggag aatggagctg tctatgggcc ccatccaggc caatcacacc      300

ggaacaggac tgctgctgac cctgcagcct gagcagaagt tccagaaggt gaagggcttt      360

ggaggagcaa tgacagacgc agccgccctg aacatcctgg ccctgagccc acccgcccag      420

aatctgctgc tgaagtctta cttcagcgag gagggcatcg gctataacat catccgggtg      480

ccaatggcct cctgtgactt ttctatcaga acctacacat atgccgatac ccccgacgat      540

ttccagctgc acaatttttc cctgcctgag gaggatacaa agctgaagat cccactgatc      600

cacagggccc tgcagctggc acagaggcct gtgagcctgc tggcatcccc atggacctct      660

cccacatggc tgaagaccaa cggcgccgtg aatggcaagg gaagcctgaa gggacagcca      720

ggcgacatct accaccagac atgggcccgg tatttcgtga agtttctgga tgcctacgcc      780

gagcacaagc tgcagttctg ggcagtgacc gcagagaacg agccatctgc cggactgctg      840

agcggatatc cattccagtg cctgggcttt acacccgagc accagaggga ctttatcgca      900

agggatctgg gacctaccct ggccaactcc acacaccaca atgtgcggct gctgatgctg      960

gacgatcaga gactgctgct gccccactgg gccaaggtgg tgctgaccga ccctgaggcc     1020

gccaagtacg tgcacggcat cgccgtgcac tggtatctgg atttcctggc accagcaaag     1080

gccaccctgg gagagacaca caggctgttc cccaacacca tgctgtttgc cagcgaggcc     1140

tgcgtgggct ccaagttttg ggagcagtcc gtgcggctgg gatcttggga cagaggcatg     1200

cagtacagcc actccatcat caccaatctg ctgtatcacg tggtgggctg gacagactgg     1260

aacctggccc tgaatcctga gggcggccca aactgggtga gaaatttcgt ggatagcccc     1320

atcatcgtgg acatcaccaa ggatacattc tacaagcagc ctatgtttta tcacctgggc     1380

cacttctcta agtttatccc agagggaagc cagagggtgg gactggtggc aagccagaag     1440

aacgacctgg atgccgtggc cctgatgcac ccagatggct ccgccgtggt ggtggtgctg     1500

aatcgctcct ctaaggacgt gcccctgacc atcaaggatc ctgccgtggg ctttctggag     1560

accatcagcc ccggctactc catccacaca tatctgtgga ggcgccagtg a              1611


<210>  14
<211>  1611
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Codon optimised sequence

<400>  14
atggagttct cttctccatc tcgcgaggag tgccctaagc cactgagtcg agtgtctatc       60

atggccggca gtctgacagg actcctcctc ctacaagctg tgtcttgggc cagtggcgcc      120

cggccatgca tcccaaagtc tttcgggtac tctagcgtgg tgtgcgtgtg caacgctaca      180

tactgcgact ctttcgaccc acctacattc cccgccctgg gaaccttcag ccgatacgag      240

tctacacggt ccgggcggcg catggagctg tcaatggggc caattcaggc taaccacacc      300

ggaacaggac tcctgctgac attacaaccc gagcagaagt tccagaaggt gaaggggttc      360

ggcggagcta tgaccgacgc tgccgctctg aacattctgg ccctgtcccc acccgctcag      420

aacctgctcc tcaagtctta cttcagcgag gagggaatcg ggtacaacat tattagagtg      480

cctatggctt cttgcgactt cagcatccgc acatacacat acgccgacac ccctgacgac      540

ttccagctgc acaacttcag cctcccagag gaggacacaa agctcaagat cccactgatt      600

caccgcgctt tacagctcgc tcagaggcca gtgtctctgc tcgcttcccc ttggacatct      660

ccaacatggt taaaaacaaa cggcgccgtg aacggaaagg ggtccctgaa ggggcagccc      720

ggagacattt accaccagac atgggccagg tacttcgtga agttcctcga cgcttacgct      780

gagcacaagc tccagttctg ggccgtgacc gccgagaacg agcctagcgc cggcctgctg      840

tccggatacc ctttccagtg cctcggattc acaccagagc accagcggga cttcattgct      900

agagacctcg ggccaacact cgctaactct acccaccaca acgtgagact gctgatgctc      960

gacgaccagc gcctcctgct gcctcactgg gctaaggtgg tgctcacaga ccccgaggcc     1020

gccaagtacg tgcacggaat tgccgtgcac tggtatctcg acttcctcgc cccagctaag     1080

gctacactcg gcgagaccca caggctgttc cctaacacaa tgctgttcgc cagcgaggct     1140

tgcgtgggaa gcaagttctg ggagcagtcc gtgagactcg gatcttggga ccgcggaatg     1200

cagtactctc actctatcat tacaaacctc ctgtaccacg tggtgggatg gaccgactgg     1260

aacctggctc tcaaccccga gggcggccca aactgggtga gaaacttcgt ggacagccct     1320

attattgtgg acattacaaa ggacaccttc tacaagcagc ctatgttcta ccacctcggg     1380

cacttcagca agttcatccc cgagggatct cagagagtgg gactggtggc ctctcagaag     1440

aacgacctcg acgctgtggc tctgatgcac cctgacgggt cggccgtggt ggtggtgctc     1500

aaccgatcta gcaaggacgt gccactgaca attaaggacc ccgccgtggg gttcctggag     1560

acaattagcc ccggatactc cattcacaca tacctgtggc gcaggcagtg a              1611


<210>  15
<211>  1611
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Codon optimised sequence encoding GBA1

<400>  15
atggagttta gcagccctag cagagaagag tgccctaagc ctctgtctcg tgtgagcatc       60

atggccggct ccttaaccgg attattactg ctgcaagctg tgtcttgggc ttccggcgct      120

aggccttgta ttcccaagtc cttcggctac tcctccgtgg tgtgcgtctg taatgccacc      180

tactgtgaca gcttcgatcc ccccacattc cccgctctgg gcacattttc tcgttacgaa      240

agcactcgta gcggaaggag gatggagctg agcatgggcc ctattcaagc taaccacacc      300

ggcaccggtt tattactgac tttacaaccc gagcagaagt tccagaaggt gaagggcttt      360

ggaggcgcca tgacagacgc cgccgcttta aatattctgg ctttaagccc tcccgcccaa      420

aatctgctgc tgaagtccta cttctccgag gaaggcatcg gctacaacat cattagggtc      480

cctatggcct cttgtgactt cagcattagg acctacacct acgccgacac ccccgacgac      540

ttccagctgc acaacttctc tttacccgag gaggatacaa agctgaagat ccctttaatc      600

catcgtgccc tccagctggc tcagaggccc gttagcttat tagcctcccc ttggacatcc      660

cctacttggc tgaaaaccaa cggcgctgtc aacggaaagg gctctttaaa gggacagccc      720

ggcgacattt accaccagac atgggctagg tacttcgtga agtttttaga cgcttatgcc      780

gaacacaagc tgcagttctg ggccgtgacc gctgagaacg agccttccgc cggtttactg      840

tccggatacc ccttccaatg tctgggattc acccccgaac accagaggga cttcatcgcc      900

agagatctgg gccccacact ggccaattcc acacaccaca acgtgagact gctcatgctg      960

gacgaccaga ggttattact ccctcactgg gccaaagtgg tgctgaccga ccccgaagct     1020

gccaagtatg tgcatggaat cgccgtgcac tggtatctgg actttctggc ccccgccaag     1080

gctacactgg gcgagaccca cagactgttc cctaacacca tgctcttcgc cagcgaagct     1140

tgcgtgggaa gcaagttctg ggagcaatcc gtgaggctgg gcagctggga tcgtggcatg     1200

cagtactccc actccattat cacaaatctg ctctaccacg tcgtgggatg gaccgattgg     1260

aatttagctc tgaaccccga gggaggcccc aattgggtga ggaacttcgt ggattccccc     1320

atcatcgtcg atatcaccaa ggacacattc tacaagcagc ccatgttcta ccatttaggc     1380

cactttagca agttcatccc cgagggaagc cagagagtgg gtttagtggc ctcccagaag     1440

aacgatctgg acgccgtcgc cctcatgcat cccgatggca gcgctgtggt cgtcgtgctg     1500

aatagaagct ccaaagacgt ccctttaacc atcaaggacc ccgccgtggg ctttctggaa     1560

accatctccc ccggttatag catccacacc tatttatgga gaagacagta a              1611


<210>  16
<211>  1611
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Codon optimised sequence encoding GBA1

<400>  16
atggaatttt ctagcccctc cagagaggaa tgccccaagc ctctgagccg ggtgtcaatc       60

atggccggat ctctgacagg actgctgctg cttcaggccg tgtcttgggc ttctggcgct      120

agaccttgca tccccaagag cttcggctac agcagcgtcg tgtgcgtgtg caatgccacc      180

tactgcgaca gcttcgaccc tcctaccttt cctgctctgg gcaccttcag cagatacgag      240

agcaccagat ccggcagacg gatggaactg agcatgggac ccatccaggc caatcacaca      300

ggcactggcc tgctgctgac actgcagccc gagcagaaat tccagaaagt gaaaggcttc      360

ggcggagcca tgacagatgc cgccgctctg aatatcctgg ctctgtctcc accagctcag      420

aacctgctgc tcaagagcta cttcagcgag gaaggcatcg gctacaacat catcagagtg      480

cccatggcca gctgcgactt cagcatcagg acctacacct acgccgacac acccgacgat      540

ttccagctgc acaacttcag cctgcctgaa gaggacacca agctgaagat ccctctgatc      600

cacagagccc tgcagctggc acaaagaccc gtttctctgc tggctagccc ctggacatct      660

cccacctggc tgaaaacaaa tggcgccgtg aatggcaagg gcagcctgaa aggccaacct      720

ggcgatatct accaccagac ctgggccaga tacttcgtga agttcctgga cgcctatgcc      780

gagcacaagc tgcagttttg ggccgtgaca gccgagaacg aaccttctgc tggactgctg      840

agcggctacc cctttcagtg cctgggcttt acacccgagc accagcggga ctttatcgcc      900

agagatctgg gacccacact ggccaatagc acccaccata atgtgcggct gctgatgctg      960

gacgaccaga gactgcttct gccccactgg gctaaagtgg tgctgacaga tcctgaggcc     1020

gccaaatacg tgcacggaat cgccgtgcac tggtatctgg actttctggc ccctgccaag     1080

gccacactgg gagagacaca cagactgttc cccaacacca tgctgttcgc cagcgaagcc     1140

tgtgtgggca gcaagttttg ggaacagagc gtgcggctcg gcagctggga tagaggcatg     1200

cagtacagcc acagcatcat caccaacctg ctgtaccacg tcgtcggctg gaccgactgg     1260

aatctggccc tgaatcctga aggcggccct aactgggtcc gaaacttcgt ggacagcccc     1320

atcatcgtgg acatcaccaa ggacaccttc tacaagcagc ccatgttcta ccacctggga     1380

cacttcagca agttcatccc cgagggctct cagcgcgttg gactggtggc cagccagaag     1440

aatgatctgg acgccgtggc tctgatgcac cctgatggat ctgctgtggt ggtggtcctg     1500

aaccggtcca gcaaagatgt gcccctgacc atcaaggatc ccgccgtggg attcctggaa     1560

acaatcagcc ctggctactc catccacacc tacctgtggc ggagacagta g              1611


<210>  17
<211>  5961
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  pAAV.CBA.hGBA1.WPRE.hGHpA

<400>  17
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 agaattcgat     1620

atcaagctta tcgataatca acctctggat tacaaaattt gtgaaagatt gactggtatt     1680

cttaactatg ttgctccttt tacgctatgt ggatacgctg ctttaatgcc tttgtatcat     1740

gctattgctt cccgtatggc tttcattttc tcctccttgt ataaatcctg gttgctgtct     1800

ctttatgagg agttgtggcc cgttgtcagg caacgtggcg tggtgtgcac tgtgtttgct     1860

gacgcaaccc ccactggttg gggcattgcc accacctgtc agctcctttc cgggactttc     1920

gctttccccc tccctattgc cacggcggaa ctcatcgccg cctgccttgc ccgctgctgg     1980

acaggggctc ggctgttggg cactgacaat tccgtggtgt tgtcggggaa atcatcgtcc     2040

tttccttggc tgctcgccta tgttgccacc tggattctgc gcgggacgtc cttctgctac     2100

gtcccttcgg ccctcaatcc agcggacctt ccttcccgcg gcctgctgcc ggctctgcgg     2160

cctcttccgc gtcttcgcct tcgccctcag acgagtcgga tctccctttg ggccgcctcc     2220

ccgcatcgat accgagcgct gctcgagaga tctacgggtg gcatccctgt gacccctccc     2280

cagtgcctct cctggccctg gaagttgcca ctccagtgcc caccagcctt gtcctaataa     2340

aattaagttg catcattttg tctgactagg tgtccttcta taatattatg gggtggaggg     2400

gggtggtatg gagcaagggg caagttggga agacaacctg tagggcctgc ggggtctatt     2460

gggaaccaag ctggagtgca gtggcacaat cttggctcac tgcaatctcc gcctcctggg     2520

ttcaagcgat tctcctgcct cagcctcccg agttgttggg attccaggca tgcatgacca     2580

ggctcagcta atttttgttt ttttggtaga gacggggttt caccatattg gccaggctgg     2640

tctccaactc ctaatctcag gtgatctacc caccttggcc tcccaaattg ctgggattac     2700

aggcgtgaac cactgctccc ttccctgtcc ttctgatttt gtaggtaacc acgtgcggac     2760

cgagcggccg caggaacccc tagtgatgga gttggccact ccctctctgc gcgctcgctc     2820

gctcactgag gccgggcgac caaaggtcgc ccgacgcccg ggctttgccc gggcggcctc     2880

agtgagcgag cgagcgcgca gctgcctgca ggggcgcctg atgcggtatt ttctccttac     2940

gcatctgtgc ggtatttcac accgcatacg tcaaagcaac catagtacgc gccctgtagc     3000

ggcgcattaa gcgcggcggg tgtggtggtt acgcgcagcg tgaccgctac acttgccagc     3060

gccttagcgc ccgctccttt cgctttcttc ccttcctttc tcgccacgtt cgccggcttt     3120

ccccgtcaag ctctaaatcg ggggctccct ttagggttcc gatttagtgc tttacggcac     3180

ctcgacccca aaaaacttga tttgggtgat ggttcacgta gtgggccatc gccctgatag     3240

acggtttttc gccctttgac gttggagtcc acgttcttta atagtggact cttgttccaa     3300

actggaacaa cactcaactc tatctcgggc tattcttttg atttataagg gattttgccg     3360

atttcggtct attggttaaa aaatgagctg atttaacaaa aatttaacgc gaattttaac     3420

aaaatattaa cgtttacaat tttatggtgc actctcagta caatctgctc tgatgccgca     3480

tagttaagcc agccccgaca cccgccaaca cccgctgacg cgccctgacg ggcttgtctg     3540

ctcccggcat ccgcttacag acaagctgtg accgtctccg ggagctgcat gtgtcagagg     3600

ttttcaccgt catcaccgaa acgcgcgaga cgaaagggcc tcgtgatacg cctattttta     3660

taggttaatg tcatgataat aatggtttct tagacgtcag gtggcacttt tcggggaaat     3720

gtgcgcggaa cccctatttg tttatttttc taaatacatt caaatatgta tccgctcatg     3780

agacaataac cctgataaat gcttcaataa tattgaaaaa ggaagagtat gagtattcaa     3840

catttccgtg tcgcccttat tccctttttt gcggcatttt gccttcctgt ttttgctcac     3900

ccagaaacgc tggtgaaagt aaaagatgct gaagatcagt tgggtgcacg agtgggttac     3960

atcgaactgg atctcaacag cggtaagatc cttgagagtt ttcgccccga agaacgtttt     4020

ccaatgatga gcacttttaa agttctgcta tgtggcgcgg tattatcccg tattgacgcc     4080

gggcaagagc aactcggtcg ccgcatacac tattctcaga atgacttggt tgagtactca     4140

ccagtcacag aaaagcatct tacggatggc atgacagtaa gagaattatg cagtgctgcc     4200

ataaccatga gtgataacac tgcggccaac ttacttctga caacgatcgg aggaccgaag     4260

gagctaaccg cttttttgca caacatgggg gatcatgtaa ctcgccttga tcgttgggaa     4320

ccggagctga atgaagccat accaaacgac gagcgtgaca ccacgatgcc tgtagcaatg     4380

gcaacaacgt tgcgcaaact attaactggc gaactactta ctctagcttc ccggcaacaa     4440

ttaatagact ggatggaggc ggataaagtt gcaggaccac ttctgcgctc ggcccttccg     4500

gctggctggt ttattgctga taaatctgga gccggtgagc gtgggtctcg cggtatcatt     4560

gcagcactgg ggccagatgg taagccctcc cgtatcgtag ttatctacac gacggggagt     4620

caggcaacta tggatgaacg aaatagacag atcgctgaga taggtgcctc actgattaag     4680

cattggtaac tgtcagacca agtttactca tatatacttt agattgattt aaaacttcat     4740

ttttaattta aaaggatcta ggtgaagatc ctttttgata atctcatgac caaaatccct     4800

taacgtgagt tttcgttcca ctgagcgtca gaccccgtag aaaagatcaa aggatcttct     4860

tgagatcctt tttttctgcg cgtaatctgc tgcttgcaaa caaaaaaacc accgctacca     4920

gcggtggttt gtttgccgga tcaagagcta ccaactcttt ttccgaaggt aactggcttc     4980

agcagagcgc agataccaaa tactgttctt ctagtgtagc cgtagttagg ccaccacttc     5040

aagaactctg tagcaccgcc tacatacctc gctctgctaa tcctgttacc agtggctgct     5100

gccagtggcg ataagtcgtg tcttaccggg ttggactcaa gacgatagtt accggataag     5160

gcgcagcggt cgggctgaac ggggggttcg tgcacacagc ccagcttgga gcgaacgacc     5220

tacaccgaac tgagatacct acagcgtgag ctatgagaaa gcgccacgct tcccgaaggg     5280

agaaaggcgg acaggtatcc ggtaagcggc agggtcggaa caggagagcg cacgagggag     5340

cttccagggg gaaacgcctg gtatctttat agtcctgtcg ggtttcgcca cctctgactt     5400

gagcgtcgat ttttgtgatg ctcgtcaggg gggcggagcc tatggaaaaa cgccagcaac     5460

gcggcctttt tacggttcct ggccttttgc tggccttttg ctcacatgtc ctgcaggcag     5520

ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt     5580

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact     5640

aggggttcct gcggccgcac gcgtccacgt tctgcttcac tctccccatc tcccccccct     5700

ccccaccccc aattttgtat ttatttattt tttaattatt ttgtgcagcg atgggggcgg     5760

gggggggggg ggcgcgcgcc aggcggggcg gggcggggcg aggggcgggg cggggcgagg     5820

cggagaggtg cggcggcagc caatcagagc ggcgcgctcc gaaagtttcc ttttatggcg     5880

aggcggcggc ggcggcggcc ctataaaaag cgaagcgcgc ggcggggtac cggatcctct     5940

agagtcgact ccggagccac c                                               5961


<210>  18
<211>  6359
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  pAAV.CAG.hGBA1.WPRE.hGHpA

<400>  18
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 agaattcgat     1620

atcaagctta tcgataatca acctctggat tacaaaattt gtgaaagatt gactggtatt     1680

cttaactatg ttgctccttt tacgctatgt ggatacgctg ctttaatgcc tttgtatcat     1740

gctattgctt cccgtatggc tttcattttc tcctccttgt ataaatcctg gttgctgtct     1800

ctttatgagg agttgtggcc cgttgtcagg caacgtggcg tggtgtgcac tgtgtttgct     1860

gacgcaaccc ccactggttg gggcattgcc accacctgtc agctcctttc cgggactttc     1920

gctttccccc tccctattgc cacggcggaa ctcatcgccg cctgccttgc ccgctgctgg     1980

acaggggctc ggctgttggg cactgacaat tccgtggtgt tgtcggggaa atcatcgtcc     2040

tttccttggc tgctcgccta tgttgccacc tggattctgc gcgggacgtc cttctgctac     2100

gtcccttcgg ccctcaatcc agcggacctt ccttcccgcg gcctgctgcc ggctctgcgg     2160

cctcttccgc gtcttcgcct tcgccctcag acgagtcgga tctccctttg ggccgcctcc     2220

ccgcatcgat accgagcgct gctcgagaga tctacgggtg gcatccctgt gacccctccc     2280

cagtgcctct cctggccctg gaagttgcca ctccagtgcc caccagcctt gtcctaataa     2340

aattaagttg catcattttg tctgactagg tgtccttcta taatattatg gggtggaggg     2400

gggtggtatg gagcaagggg caagttggga agacaacctg tagggcctgc ggggtctatt     2460

gggaaccaag ctggagtgca gtggcacaat cttggctcac tgcaatctcc gcctcctggg     2520

ttcaagcgat tctcctgcct cagcctcccg agttgttggg attccaggca tgcatgacca     2580

ggctcagcta atttttgttt ttttggtaga gacggggttt caccatattg gccaggctgg     2640

tctccaactc ctaatctcag gtgatctacc caccttggcc tcccaaattg ctgggattac     2700

aggcgtgaac cactgctccc ttccctgtcc ttctgatttt gtaggtaacc acgtgcggac     2760

cgagcggccg caggaacccc tagtgatgga gttggccact ccctctctgc gcgctcgctc     2820

gctcactgag gccgggcgac caaaggtcgc ccgacgcccg ggctttgccc gggcggcctc     2880

agtgagcgag cgagcgcgca gctgcctgca ggggcgcctg atgcggtatt ttctccttac     2940

gcatctgtgc ggtatttcac accgcatacg tcaaagcaac catagtacgc gccctgtagc     3000

ggcgcattaa gcgcggcggg tgtggtggtt acgcgcagcg tgaccgctac acttgccagc     3060

gccttagcgc ccgctccttt cgctttcttc ccttcctttc tcgccacgtt cgccggcttt     3120

ccccgtcaag ctctaaatcg ggggctccct ttagggttcc gatttagtgc tttacggcac     3180

ctcgacccca aaaaacttga tttgggtgat ggttcacgta gtgggccatc gccctgatag     3240

acggtttttc gccctttgac gttggagtcc acgttcttta atagtggact cttgttccaa     3300

actggaacaa cactcaactc tatctcgggc tattcttttg atttataagg gattttgccg     3360

atttcggtct attggttaaa aaatgagctg atttaacaaa aatttaacgc gaattttaac     3420

aaaatattaa cgtttacaat tttatggtgc actctcagta caatctgctc tgatgccgca     3480

tagttaagcc agccccgaca cccgccaaca cccgctgacg cgccctgacg ggcttgtctg     3540

ctcccggcat ccgcttacag acaagctgtg accgtctccg ggagctgcat gtgtcagagg     3600

ttttcaccgt catcaccgaa acgcgcgaga cgaaagggcc tcgtgatacg cctattttta     3660

taggttaatg tcatgataat aatggtttct tagacgtcag gtggcacttt tcggggaaat     3720

gtgcgcggaa cccctatttg tttatttttc taaatacatt caaatatgta tccgctcatg     3780

agacaataac cctgataaat gcttcaataa tattgaaaaa ggaagagtat gagtattcaa     3840

catttccgtg tcgcccttat tccctttttt gcggcatttt gccttcctgt ttttgctcac     3900

ccagaaacgc tggtgaaagt aaaagatgct gaagatcagt tgggtgcacg agtgggttac     3960

atcgaactgg atctcaacag cggtaagatc cttgagagtt ttcgccccga agaacgtttt     4020

ccaatgatga gcacttttaa agttctgcta tgtggcgcgg tattatcccg tattgacgcc     4080

gggcaagagc aactcggtcg ccgcatacac tattctcaga atgacttggt tgagtactca     4140

ccagtcacag aaaagcatct tacggatggc atgacagtaa gagaattatg cagtgctgcc     4200

ataaccatga gtgataacac tgcggccaac ttacttctga caacgatcgg aggaccgaag     4260

gagctaaccg cttttttgca caacatgggg gatcatgtaa ctcgccttga tcgttgggaa     4320

ccggagctga atgaagccat accaaacgac gagcgtgaca ccacgatgcc tgtagcaatg     4380

gcaacaacgt tgcgcaaact attaactggc gaactactta ctctagcttc ccggcaacaa     4440

ttaatagact ggatggaggc ggataaagtt gcaggaccac ttctgcgctc ggcccttccg     4500

gctggctggt ttattgctga taaatctgga gccggtgagc gtgggtctcg cggtatcatt     4560

gcagcactgg ggccagatgg taagccctcc cgtatcgtag ttatctacac gacggggagt     4620

caggcaacta tggatgaacg aaatagacag atcgctgaga taggtgcctc actgattaag     4680

cattggtaac tgtcagacca agtttactca tatatacttt agattgattt aaaacttcat     4740

ttttaattta aaaggatcta ggtgaagatc ctttttgata atctcatgac caaaatccct     4800

taacgtgagt tttcgttcca ctgagcgtca gaccccgtag aaaagatcaa aggatcttct     4860

tgagatcctt tttttctgcg cgtaatctgc tgcttgcaaa caaaaaaacc accgctacca     4920

gcggtggttt gtttgccgga tcaagagcta ccaactcttt ttccgaaggt aactggcttc     4980

agcagagcgc agataccaaa tactgttctt ctagtgtagc cgtagttagg ccaccacttc     5040

aagaactctg tagcaccgcc tacatacctc gctctgctaa tcctgttacc agtggctgct     5100

gccagtggcg ataagtcgtg tcttaccggg ttggactcaa gacgatagtt accggataag     5160

gcgcagcggt cgggctgaac ggggggttcg tgcacacagc ccagcttgga gcgaacgacc     5220

tacaccgaac tgagatacct acagcgtgag ctatgagaaa gcgccacgct tcccgaaggg     5280

agaaaggcgg acaggtatcc ggtaagcggc agggtcggaa caggagagcg cacgagggag     5340

cttccagggg gaaacgcctg gtatctttat agtcctgtcg ggtttcgcca cctctgactt     5400

gagcgtcgat ttttgtgatg ctcgtcaggg gggcggagcc tatggaaaaa cgccagcaac     5460

gcggcctttt tacggttcct ggccttttgc tggccttttg ctcacatgtc ctgcaggcag     5520

ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt     5580

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact     5640

aggggttcct gcggccgcac gcgtgacatt gattattgac tagttattaa tagtaatcaa     5700

ttacggggtc attagttcat agcccatata tggagttccg cgttacataa cttacggtaa     5760

atggcccgcc tggctgaccg cccaacgacc cccgcccatt gacgtcaata atgacgtatg     5820

ttcccatagt aacgccaata gggactttcc attgacgtca atgggtggag tatttacggt     5880

aaactgccca cttggcagta catcaagtgt atcatatgcc aagtacgccc cctattgacg     5940

tcaatgacgg taaatggccc gcctggcatt atgcccagta catgacctta tgggactttc     6000

ctacttggca gtacatctac gtattagtca tcgctattac catggtcgag gtgagcccca     6060

cgttctgctt cactctcccc atctcccccc cctccccacc cccaattttg tatttattta     6120

ttttttaatt attttgtgca gcgatggggg cggggggggg gggggggcgc gcgccaggcg     6180

gggcggggcg gggcgagggg cggggcgggg cgaggcggag aggtgcggcg gcagccaatc     6240

agagcggcgc gctccgaaag tttcctttta tggcgaggcg gcggcggcgg cggccctata     6300

aaaagcgaag cgcgcggcgg gcgggtaccg gatcctctag agtcgactcc ggagccacc      6359


<210>  19
<211>  6185
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  pAAV.hSYN.hGBA1.WPRE.hGHpA

<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 agaattcgat     1620

atcaagctta tcgataatca acctctggat tacaaaattt gtgaaagatt gactggtatt     1680

cttaactatg ttgctccttt tacgctatgt ggatacgctg ctttaatgcc tttgtatcat     1740

gctattgctt cccgtatggc tttcattttc tcctccttgt ataaatcctg gttgctgtct     1800

ctttatgagg agttgtggcc cgttgtcagg caacgtggcg tggtgtgcac tgtgtttgct     1860

gacgcaaccc ccactggttg gggcattgcc accacctgtc agctcctttc cgggactttc     1920

gctttccccc tccctattgc cacggcggaa ctcatcgccg cctgccttgc ccgctgctgg     1980

acaggggctc ggctgttggg cactgacaat tccgtggtgt tgtcggggaa atcatcgtcc     2040

tttccttggc tgctcgccta tgttgccacc tggattctgc gcgggacgtc cttctgctac     2100

gtcccttcgg ccctcaatcc agcggacctt ccttcccgcg gcctgctgcc ggctctgcgg     2160

cctcttccgc gtcttcgcct tcgccctcag acgagtcgga tctccctttg ggccgcctcc     2220

ccgcatcgat accgagcgct gctcgagaga tctacgggtg gcatccctgt gacccctccc     2280

cagtgcctct cctggccctg gaagttgcca ctccagtgcc caccagcctt gtcctaataa     2340

aattaagttg catcattttg tctgactagg tgtccttcta taatattatg gggtggaggg     2400

gggtggtatg gagcaagggg caagttggga agacaacctg tagggcctgc ggggtctatt     2460

gggaaccaag ctggagtgca gtggcacaat cttggctcac tgcaatctcc gcctcctggg     2520

ttcaagcgat tctcctgcct cagcctcccg agttgttggg attccaggca tgcatgacca     2580

ggctcagcta atttttgttt ttttggtaga gacggggttt caccatattg gccaggctgg     2640

tctccaactc ctaatctcag gtgatctacc caccttggcc tcccaaattg ctgggattac     2700

aggcgtgaac cactgctccc ttccctgtcc ttctgatttt gtaggtaacc acgtgcggac     2760

cgagcggccg caggaacccc tagtgatgga gttggccact ccctctctgc gcgctcgctc     2820

gctcactgag gccgggcgac caaaggtcgc ccgacgcccg ggctttgccc gggcggcctc     2880

agtgagcgag cgagcgcgca gctgcctgca ggggcgcctg atgcggtatt ttctccttac     2940

gcatctgtgc ggtatttcac accgcatacg tcaaagcaac catagtacgc gccctgtagc     3000

ggcgcattaa gcgcggcggg tgtggtggtt acgcgcagcg tgaccgctac acttgccagc     3060

gccttagcgc ccgctccttt cgctttcttc ccttcctttc tcgccacgtt cgccggcttt     3120

ccccgtcaag ctctaaatcg ggggctccct ttagggttcc gatttagtgc tttacggcac     3180

ctcgacccca aaaaacttga tttgggtgat ggttcacgta gtgggccatc gccctgatag     3240

acggtttttc gccctttgac gttggagtcc acgttcttta atagtggact cttgttccaa     3300

actggaacaa cactcaactc tatctcgggc tattcttttg atttataagg gattttgccg     3360

atttcggtct attggttaaa aaatgagctg atttaacaaa aatttaacgc gaattttaac     3420

aaaatattaa cgtttacaat tttatggtgc actctcagta caatctgctc tgatgccgca     3480

tagttaagcc agccccgaca cccgccaaca cccgctgacg cgccctgacg ggcttgtctg     3540

ctcccggcat ccgcttacag acaagctgtg accgtctccg ggagctgcat gtgtcagagg     3600

ttttcaccgt catcaccgaa acgcgcgaga cgaaagggcc tcgtgatacg cctattttta     3660

taggttaatg tcatgataat aatggtttct tagacgtcag gtggcacttt tcggggaaat     3720

gtgcgcggaa cccctatttg tttatttttc taaatacatt caaatatgta tccgctcatg     3780

agacaataac cctgataaat gcttcaataa tattgaaaaa ggaagagtat gagtattcaa     3840

catttccgtg tcgcccttat tccctttttt gcggcatttt gccttcctgt ttttgctcac     3900

ccagaaacgc tggtgaaagt aaaagatgct gaagatcagt tgggtgcacg agtgggttac     3960

atcgaactgg atctcaacag cggtaagatc cttgagagtt ttcgccccga agaacgtttt     4020

ccaatgatga gcacttttaa agttctgcta tgtggcgcgg tattatcccg tattgacgcc     4080

gggcaagagc aactcggtcg ccgcatacac tattctcaga atgacttggt tgagtactca     4140

ccagtcacag aaaagcatct tacggatggc atgacagtaa gagaattatg cagtgctgcc     4200

ataaccatga gtgataacac tgcggccaac ttacttctga caacgatcgg aggaccgaag     4260

gagctaaccg cttttttgca caacatgggg gatcatgtaa ctcgccttga tcgttgggaa     4320

ccggagctga atgaagccat accaaacgac gagcgtgaca ccacgatgcc tgtagcaatg     4380

gcaacaacgt tgcgcaaact attaactggc gaactactta ctctagcttc ccggcaacaa     4440

ttaatagact ggatggaggc ggataaagtt gcaggaccac ttctgcgctc ggcccttccg     4500

gctggctggt ttattgctga taaatctgga gccggtgagc gtgggtctcg cggtatcatt     4560

gcagcactgg ggccagatgg taagccctcc cgtatcgtag ttatctacac gacggggagt     4620

caggcaacta tggatgaacg aaatagacag atcgctgaga taggtgcctc actgattaag     4680

cattggtaac tgtcagacca agtttactca tatatacttt agattgattt aaaacttcat     4740

ttttaattta aaaggatcta ggtgaagatc ctttttgata atctcatgac caaaatccct     4800

taacgtgagt tttcgttcca ctgagcgtca gaccccgtag aaaagatcaa aggatcttct     4860

tgagatcctt tttttctgcg cgtaatctgc tgcttgcaaa caaaaaaacc accgctacca     4920

gcggtggttt gtttgccgga tcaagagcta ccaactcttt ttccgaaggt aactggcttc     4980

agcagagcgc agataccaaa tactgttctt ctagtgtagc cgtagttagg ccaccacttc     5040

aagaactctg tagcaccgcc tacatacctc gctctgctaa tcctgttacc agtggctgct     5100

gccagtggcg ataagtcgtg tcttaccggg ttggactcaa gacgatagtt accggataag     5160

gcgcagcggt cgggctgaac ggggggttcg tgcacacagc ccagcttgga gcgaacgacc     5220

tacaccgaac tgagatacct acagcgtgag ctatgagaaa gcgccacgct tcccgaaggg     5280

agaaaggcgg acaggtatcc ggtaagcggc agggtcggaa caggagagcg cacgagggag     5340

cttccagggg gaaacgcctg gtatctttat agtcctgtcg ggtttcgcca cctctgactt     5400

gagcgtcgat ttttgtgatg ctcgtcaggg gggcggagcc tatggaaaaa cgccagcaac     5460

gcggcctttt tacggttcct ggccttttgc tggccttttg ctcacatgtc ctgcaggcag     5520

ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt     5580

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact     5640

aggggttcct gcggccgcac gcgtgtgtct agactgcaga gggccctgcg tatgagtgca     5700

agtgggtttt aggaccagga tgaggcgggg tgggggtgcc tacctgacga ccgaccccga     5760

cccactggac aagcacccaa cccccattcc ccaaattgcg catcccctat cagagagggg     5820

gaggggaaac aggatgcggc gaggcgcgtg cgcactgcca gcttcagcac cgcggacagt     5880

gccttcgccc ccgcctggcg gcgcgcgcca ccgccgcctc agcactgaag gcgcgctgac     5940

gtcactcgcc ggtcccccgc aaactcccct tcccggccac cttggtcgcg tccgcgccgc     6000

cgccggccca gccggaccgc accacgcgag gcgcgagata ggggggcacg ggcgcgacca     6060

tctgcgctgc ggcgccggcg actcagcgct gcctcagtct gcggtgggca gcggaggagt     6120

cgtgtcgtgc ctgagagcgc agtcgagaag gtaccggatc ctctagagtc gactccggag     6180

ccacc                                                                 6185


<210>  20
<211>  2290
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CAG GBA1 expression construct

<400>  20
tagttattaa tagtaatcaa ttacggggtc attagttcat agcccatata tggagttccg       60

cgttacataa cttacggtaa atggcccgcc tggctgaccg cccaacgacc cccgcccatt      120

gacgtcaata atgacgtatg ttcccatagt aacgccaata gggactttcc attgacgtca      180

atgggtggag tatttacggt aaactgccca cttggcagta catcaagtgt atcatatgcc      240

aagtacgccc cctattgacg tcaatgacgg taaatggccc gcctggcatt atgcccagta      300

catgacctta tgggactttc ctacttggca gtacatctac gtattagtca tcgctattac      360

catggtcgag gtgagcccca cgttctgctt cactctcccc atctcccccc cctccccacc      420

cccaattttg tatttattta ttttttaatt attttgtgca gcgatggggg cggggggggg      480

gggggggcgc gcgccaggcg gggcggggcg gggcgagggg cggggcgggg cgaggcggag      540

aggtgcggcg gcagccaatc agagcggcgc gctccgaaag tttcctttta tggcgaggcg      600

gcggcggcgg cggccctata aaaagcgaag cgcgcggcgg gcgggtaccg gatcctctag      660

agtcgactcc ggagccacca tggagttttc aagtccttcc agagaggaat gtcccaagcc      720

tttgagtagg gtaagcatca tggctggcag cctcacagga ttgcttctac ttcaggcagt      780

gtcgtgggca tcaggtgccc gcccctgcat ccctaaaagc ttcggctaca gctcggtggt      840

gtgtgtctgc aatgccacat actgtgactc ctttgacccc ccgacctttc ctgcccttgg      900

taccttcagc cgctatgaga gtacacgcag tgggcgacgg atggagctga gtatggggcc      960

catccaggct aatcacacgg gcacaggcct gctactgacc ctgcagccag aacagaagtt     1020

ccagaaagtg aagggatttg gaggggccat gacagatgct gctgctctca acatccttgc     1080

cctgtcaccc cctgcccaaa atttgctact taaatcgtac ttctctgaag aaggaatcgg     1140

atataacatc atccgggtac ccatggccag ctgtgacttc tccatccgca cctacaccta     1200

tgcagacacc cctgatgatt tccagttgca caacttcagc ctcccagagg aagataccaa     1260

gctcaagata cccctgattc accgagccct gcagttggcc cagcgtcccg tttcactcct     1320

tgccagcccc tggacatcac ccacttggct caagaccaat ggagcggtga atgggaaggg     1380

gtcactcaag ggacagcccg gagacatcta ccaccagacc tgggccagat actttgtgaa     1440

gttcctggat gcctatgctg agcacaagtt acagttctgg gcagtgacag ctgaaaatga     1500

gccttctgct gggctgttga gtggataccc cttccagtgc ctgggcttca cccctgaaca     1560

tcagcgagac ttcattgccc gtgacctagg tcctaccctc gccaacagta ctcaccacaa     1620

tgtccgccta ctcatgctgg atgaccaacg cttgctgctg ccccactggg caaaggtggt     1680

actgacagac ccagaagcag ctaaatatgt tcatggcatt gctgtacatt ggtacctgga     1740

ctttctggct ccagccaaag ccaccctagg ggagacacac cgcctgttcc ccaacaccat     1800

gctctttgcc tcagaggcct gtgtgggctc caagttctgg gagcagagtg tgcggctagg     1860

ctcctgggat cgagggatgc agtacagcca cagcatcatc acgaacctcc tgtaccatgt     1920

ggtcggctgg accgactgga accttgccct gaaccccgaa ggaggaccca attgggtgcg     1980

taactttgtc gacagtccca tcattgtaga catcaccaag gacacgtttt acaaacagcc     2040

catgttctac caccttggcc acttcagcaa gttcattcct gagggctccc agagagtggg     2100

gctggttgcc agtcagaaga acgacctgga cgcagtggca ctgatgcatc ccgatggctc     2160

tgctgttgtg gtcgtgctaa accgctcctc taaggatgtg cctcttacca tcaaggatcc     2220

tgctgtgggc ttcctggaga caatctcacc tggctactcc attcacacct acctgtggcg     2280

tcgccagtga                                                            2290


<210>  21
<211>  2913
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CAG GBA1 WPRE expression construct

<400>  21
tagttattaa tagtaatcaa ttacggggtc attagttcat agcccatata tggagttccg       60

cgttacataa cttacggtaa atggcccgcc tggctgaccg cccaacgacc cccgcccatt      120

gacgtcaata atgacgtatg ttcccatagt aacgccaata gggactttcc attgacgtca      180

atgggtggag tatttacggt aaactgccca cttggcagta catcaagtgt atcatatgcc      240

aagtacgccc cctattgacg tcaatgacgg taaatggccc gcctggcatt atgcccagta      300

catgacctta tgggactttc ctacttggca gtacatctac gtattagtca tcgctattac      360

catggtcgag gtgagcccca cgttctgctt cactctcccc atctcccccc cctccccacc      420

cccaattttg tatttattta ttttttaatt attttgtgca gcgatggggg cggggggggg      480

gggggggcgc gcgccaggcg gggcggggcg gggcgagggg cggggcgggg cgaggcggag      540

aggtgcggcg gcagccaatc agagcggcgc gctccgaaag tttcctttta tggcgaggcg      600

gcggcggcgg cggccctata aaaagcgaag cgcgcggcgg gcgggtaccg gatcctctag      660

agtcgactcc ggagccacca tggagttttc aagtccttcc agagaggaat gtcccaagcc      720

tttgagtagg gtaagcatca tggctggcag cctcacagga ttgcttctac ttcaggcagt      780

gtcgtgggca tcaggtgccc gcccctgcat ccctaaaagc ttcggctaca gctcggtggt      840

gtgtgtctgc aatgccacat actgtgactc ctttgacccc ccgacctttc ctgcccttgg      900

taccttcagc cgctatgaga gtacacgcag tgggcgacgg atggagctga gtatggggcc      960

catccaggct aatcacacgg gcacaggcct gctactgacc ctgcagccag aacagaagtt     1020

ccagaaagtg aagggatttg gaggggccat gacagatgct gctgctctca acatccttgc     1080

cctgtcaccc cctgcccaaa atttgctact taaatcgtac ttctctgaag aaggaatcgg     1140

atataacatc atccgggtac ccatggccag ctgtgacttc tccatccgca cctacaccta     1200

tgcagacacc cctgatgatt tccagttgca caacttcagc ctcccagagg aagataccaa     1260

gctcaagata cccctgattc accgagccct gcagttggcc cagcgtcccg tttcactcct     1320

tgccagcccc tggacatcac ccacttggct caagaccaat ggagcggtga atgggaaggg     1380

gtcactcaag ggacagcccg gagacatcta ccaccagacc tgggccagat actttgtgaa     1440

gttcctggat gcctatgctg agcacaagtt acagttctgg gcagtgacag ctgaaaatga     1500

gccttctgct gggctgttga gtggataccc cttccagtgc ctgggcttca cccctgaaca     1560

tcagcgagac ttcattgccc gtgacctagg tcctaccctc gccaacagta ctcaccacaa     1620

tgtccgccta ctcatgctgg atgaccaacg cttgctgctg ccccactggg caaaggtggt     1680

actgacagac ccagaagcag ctaaatatgt tcatggcatt gctgtacatt ggtacctgga     1740

ctttctggct ccagccaaag ccaccctagg ggagacacac cgcctgttcc ccaacaccat     1800

gctctttgcc tcagaggcct gtgtgggctc caagttctgg gagcagagtg tgcggctagg     1860

ctcctgggat cgagggatgc agtacagcca cagcatcatc acgaacctcc tgtaccatgt     1920

ggtcggctgg accgactgga accttgccct gaaccccgaa ggaggaccca attgggtgcg     1980

taactttgtc gacagtccca tcattgtaga catcaccaag gacacgtttt acaaacagcc     2040

catgttctac caccttggcc acttcagcaa gttcattcct gagggctccc agagagtggg     2100

gctggttgcc agtcagaaga acgacctgga cgcagtggca ctgatgcatc ccgatggctc     2160

tgctgttgtg gtcgtgctaa accgctcctc taaggatgtg cctcttacca tcaaggatcc     2220

tgctgtgggc ttcctggaga caatctcacc tggctactcc attcacacct acctgtggcg     2280

tcgccagtga gaattcgata tcaagcttat cgataatcaa cctctggatt acaaaatttg     2340

tgaaagattg actggtattc ttaactatgt tgctcctttt acgctatgtg gatacgctgc     2400

tttaatgcct ttgtatcatg ctattgcttc ccgtatggct ttcattttct cctccttgta     2460

taaatcctgg ttgctgtctc tttatgagga gttgtggccc gttgtcaggc aacgtggcgt     2520

ggtgtgcact gtgtttgctg acgcaacccc cactggttgg ggcattgcca ccacctgtca     2580

gctcctttcc gggactttcg ctttccccct ccctattgcc acggcggaac tcatcgccgc     2640

ctgccttgcc cgctgctgga caggggctcg gctgttgggc actgacaatt ccgtggtgtt     2700

gtcggggaaa tcatcgtcct ttccttggct gctcgcctat gttgccacct ggattctgcg     2760

cgggacgtcc ttctgctacg tcccttcggc cctcaatcca gcggaccttc cttcccgcgg     2820

cctgctgccg gctctgcggc ctcttccgcg tcttcgcctt cgccctcaga cgagtcggat     2880

ctccctttgg gccgcctccc cgcatcgata ccg                                  2913


