                         SEQUENCE LISTING

<110>  Vrije Universiteit Brussel
 
<120>  NOVEL COMBINATION OF NUCLEIC ACID REGULATORY ELEMENTS AND METHODS
        AND USES THEREOF

<130>  VUB-084-PCT

<150>  20158064.4
<151>  2020-02-18

<160>  23    

<170>  PatentIn version 3.5

<210>  1
<211>  452
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Dph-CRE02

<400>  1
gacaggtgcg gttcccggag cgcaggcgca cacatgcacc caccggcgaa cgcggtgacc       60

ctcgccccac cccatcccct ccggcgggca actgggtcgg gtcaggaggg gcaaacccgc      120

tagggagaca ctccatatac ggcccggccc gcgttacctg ggaccgggcc aacccgctcc      180

ttctttggtc aacgcagggg acccgggcgg gggcccaggc cgcgaaccgg ccgagggagg      240

gggctctagt gcccaacacc caaatatggc tcgagaaggg cagcgacatt cctgcggggt      300

ggcgcggagg gaatgcccgc gggctatata aaacctgagc agagggacaa gcggccaccg      360

cagcggacag cgccaagtga agcctcgctt cccctccgcg gcgaccaggg cccgagccga      420

gagtagcagt tgtagctacc cgcccaggta gg                                    452


<210>  2
<211>  381
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CSKSH1

<400>  2
gttctcctct ataaataccc gctctggtat ttggggttgg cagctgttgc tgccagggag       60

atggttgggt tgacatgcgg ctcctgacaa aacacaaacc cctggtgtgt gtgggcgtgg      120

gtggtgtgag tagggggatg aatcagggag ggggcggggg acccaggggg caggagccac      180

acaaagtctg tgcgggggtg ggagcgcaca tagcaattgg aaactgaaag cttatcagac      240

cctttctgga aatcagccca ctgtttataa acttgaggcc ccaccctcga cagtaccggg      300

gaggaagagg gcctgcacta gtccagaggg aaactgaggc tcagggctag ctcgcccata      360

gacatacatg gcaggcaggc t                                                381


<210>  3
<211>  847
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Dph-CRE02-CSKSH1

<400>  3
gacaggtgcg gttcccggag cgcaggcgca cacatgcacc caccggcgaa cgcggtgacc       60

ctcgccccac cccatcccct ccggcgggca actgggtcgg gtcaggaggg gcaaacccgc      120

tagggagaca ctccatatac ggcccggccc gcgttacctg ggaccgggcc aacccgctcc      180

ttctttggtc aacgcagggg acccgggcgg gggcccaggc cgcgaaccgg ccgagggagg      240

gggctctagt gcccaacacc caaatatggc tcgagaaggg cagcgacatt cctgcggggt      300

ggcgcggagg gaatgcccgc gggctatata aaacctgagc agagggacaa gcggccaccg      360

cagcggacag cgccaagtga agcctcgctt cccctccgcg gcgaccaggg cccgagccga      420

gagtagcagt tgtagctacc cgcccaggta ggggcgcgcc acgcgtgttc tcctctataa      480

atacccgctc tggtatttgg ggttggcagc tgttgctgcc agggagatgg ttgggttgac      540

atgcggctcc tgacaaaaca caaacccctg gtgtgtgtgg gcgtgggtgg tgtgagtagg      600

gggatgaatc agggaggggg cgggggaccc agggggcagg agccacacaa agtctgtgcg      660

ggggtgggag cgcacatagc aattggaaac tgaaagctta tcagaccctt tctggaaatc      720

agcccactgt ttataaactt gaggccccac cctcgacagt accggggagg aagagggcct      780

gcactagtcc agagggaaac tgaggctcag ggctagctcg cccatagaca tacatggcag      840

gcaggct                                                                847


<210>  4
<211>  334
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  SPc5-12

<400>  4
tggccaccgc cttcggcacc atcctcacga cacccaaata tggcgacggg tgaggaatgg       60

tggggagtta tttttagagc ggtgaggaag gtgggcaggc agcaggtgtt ggcgctctaa      120

aaataactcc cgggagttat ttttagagcg gaggaatggt ggacacccaa atatggcgac      180

ggttcctcac ccgtcgccat atttgggtgt ccgccctcgg ccggggccgc attcctgggg      240

gccgggcggt gctcccgccc gcctcgataa aaggctccgg ggccggcggc ggcccacgag      300

ctacccggag gagcgggagg cgccaagctc taga                                  334


<210>  5
<211>  2859
<212>  DNA
<213>  Homo sapiens

<400>  5
atgggagtga ggcacccgcc ctgctcccac cggctcctgg ccgtctgcgc cctcgtgtcc       60

ttggcaaccg ctgcactcct ggggcacatc ctactccatg atttcctgct ggttccccga      120

gagctgagtg gctcctcccc agtcctggag gagactcacc cagctcacca gcagggagcc      180

agcagaccag ggccccggga tgcccaggca caccccggcc gtcccagagc agtgcccaca      240

cagtgcgacg tcccccccaa cagccgcttc gattgcgccc ctgacaaggc catcacccag      300

gaacagtgcg aggcccgcgg ctgttgctac atccctgcaa agcaggggct gcagggagcc      360

cagatggggc agccctggtg cttcttccca cccagctacc ccagctacaa gctggagaac      420

ctgagctcct ctgaaatggg ctacacggcc accctgaccc gtaccacccc caccttcttc      480

cccaaggaca tcctgaccct gcggctggac gtgatgatgg agactgagaa ccgcctccac      540

ttcacgatca aagatccagc taacaggcgc tacgaggtgc ccttggagac cccgcatgtc      600

cacagccggg caccgtcccc actctacagc gtggagttct ccgaggagcc cttcggggtg      660

atcgtgcgcc ggcagctgga cggccgcgtg ctgctgaaca cgacggtggc gcccctgttc      720

tttgcggacc agttccttca gctgtccacc tcgctgccct cgcagtatat cacaggcctc      780

gccgagcacc tcagtcccct gatgctcagc accagctgga ccaggatcac cctgtggaac      840

cgggaccttg cgcccacgcc cggtgcgaac ctctacgggt ctcacccttt ctacctggcg      900

ctggaggacg gcgggtcggc acacggggtg ttcctgctaa acagcaatgc catggatgtg      960

gtcctgcagc cgagccctgc ccttagctgg aggtcgacag gtgggatcct ggatgtctac     1020

atcttcctgg gcccagagcc caagagcgtg gtgcagcagt acctggacgt tgtgggatac     1080

ccgttcatgc cgccatactg gggcctgggc ttccacctgt gccgctgggg ctactcctcc     1140

accgctatca cccgccaggt ggtggagaac atgaccaggg cccacttccc cctggacgtc     1200

cagtggaacg acctggacta catggactcc cggagggact tcacgttcaa caaggatggc     1260

ttccgggact tcccggccat ggtgcaggag ctgcaccagg gcggccggcg ctacatgatg     1320

atcgtggatc ctgccatcag cagctcgggc cctgccggga gctacaggcc ctacgacgag     1380

ggtctgcgga ggggggtttt catcaccaac gagaccggcc agccgctgat tgggaaggta     1440

tggcccgggt ccactgcctt ccccgacttc accaacccca cagccctggc ctggtgggag     1500

gacatggtgg ctgagttcca tgaccaggtg cccttcgacg gcatgtggat tgacatgaac     1560

gagccttcca acttcatcag gggctctgag gacggctgcc ccaacaatga gctggagaac     1620

ccaccctacg tgcctggggt ggttgggggg accctccagg cggccaccat ctgtgcctcc     1680

agccaccagt ttctctccac acactacaac ctgcacaacc tctacggcct gaccgaagcc     1740

atcgcctccc acagggcgct ggtgaaggct cgggggacac gcccatttgt gatctcccgc     1800

tcgacctttg ctggccacgg ccgatacgcc ggccactgga cgggggacgt gtggagctcc     1860

tgggagcagc tcgcctcctc cgtgccagaa atcctgcagt ttaacctgct gggggtgcct     1920

ctggtcgggg ccgacgtctg cggcttcctg ggcaacacct cagaggagct gtgtgtgcgc     1980

tggacccagc tgggggcctt ctaccccttc atgcggaacc acaacagcct gctcagtctg     2040

ccccaggagc cgtacagctt cagcgagccg gcccagcagg ccatgaggaa ggccctcacc     2100

ctgcgctacg cactcctccc ccacctctac acactgttcc accaggccca cgtcgcgggg     2160

gagaccgtgg cccggcccct cttcctggag ttccccaagg actctagcac ctggactgtg     2220

gaccaccagc tcctgtgggg ggaggccctg ctcatcaccc cagtgctcca ggccgggaag     2280

gccgaagtga ctggctactt ccccttgggc acatggtacg acctgcagac ggtgccagta     2340

gaggcccttg gcagcctccc acccccacct gcagctcccc gtgagccagc catccacagc     2400

gaggggcagt gggtgacgct gccggccccc ctggacacca tcaacgtcca cctccgggct     2460

gggtacatca tccccctgca gggccctggc ctcacaacca cagagtcccg ccagcagccc     2520

atggccctgg ctgtggccct gaccaagggt ggggaggccc gaggggagct gttctgggac     2580

gatggagaga gcctggaagt gctggagcga ggggcctaca cacaggtcat cttcctggcc     2640

aggaataaca cgatcgtgaa tgagctggta cgtgtgacca gtgagggagc tggcctgcag     2700

ctgcagaagg tgactgtcct gggcgtggcc acggcgcccc agcaggtcct ctccaacggt     2760

gtccctgtct ccaacttcac ctacagcccc gacaccaagg tcctggacat ctgtgtctcg     2820

ctgttgatgg gagagcagtt tctcgtcagc tggtgttag                            2859


<210>  6
<211>  2859
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Codon optimized human acid alpha-glucosidase (hGAAco)

<400>  6
atgggcgtca gacatcctcc atgttctcac agactgctgg ccgtgtgtgc tctggtgtct       60

cttgctacag ctgccctgct gggacatatc ctgctgcacg attttctgct ggtgcccaga      120

gagctgtctg gcagctctcc tgtgctggaa gaaacacacc ctgcacatca gcagggcgcc      180

tctagacctg gacctagaga tgctcaagcc catcctggca gacctagagc cgtgcctaca      240

cagtgtgacg tgccacctaa cagcagattc gactgcgccc ctgacaaggc catcacacaa      300

gagcagtgtg aagccagagg ctgctgctac attcctgcca aacaaggact gcagggcgct      360

cagatgggac agccttggtg cttcttccca ccatcttacc ccagctacaa gctggaaaac      420

ctgagcagca gcgagatggg ctacaccgcc acactgacca gaaccacacc tacattcttc      480

ccaaaggaca tcctgacact gcggctggac gtgatgatgg aaaccgagaa ccggctgcac      540

ttcaccatca aggaccccgc caatagaaga tacgaggtgc ccctggaaac ccctcacgtg      600

cactctagag ccccatctcc actgtacagc gtggaattca gcgaggaacc ctttggcgtg      660

atcgtgcgga gacagctgga tggcagagtg ctgctgaata ccacagtggc ccctctgttc      720

ttcgccgacc agtttctgca gctgagcaca agcctgccta gccagtatat cacaggcctg      780

gccgaacacc tgtctccact gatgctgagc accagctgga ccagaatcac cctgtggaac      840

agagatctgg cccctacacc tggcgccaat ctgtacggct ctcacccttt ttatctggcc      900

ctggaagatg gcggaagcgc ccacggtgtc tttctgctga acagcaacgc catggacgtg      960

gtgctgcaac catctcctgc tctgtcttgg agaagcaccg gcggcatcct ggacgtgtac     1020

atctttctgg gacccgagcc taagagcgtg gtgcagcagt atctggatgt cgtgggctac     1080

cccttcatgc ctccttattg gggcctgggc ttccacctgt gtagatgggg atacagctcc     1140

accgccatca ccagacaggt ggtggaaaac atgacccggg ctcacttccc actggatgtg     1200

cagtggaacg acctggacta catggactcc agacgggact tcacctttaa caaggacggc     1260

ttcagagact tccccgccat ggtgcaagaa ctgcatcaag gcggcagacg gtacatgatg     1320

atcgtggatc ctgccatctc ttctagcggc cctgccggaa gctacagacc ttatgatgag     1380

ggcctgagaa gaggcgtgtt catcaccaat gagacaggcc agcctctgat cggcaaagtg     1440

tggcctggaa gcaccgcctt tccagacttc accaatccaa ccgctctggc ttggtgggaa     1500

gatatggtgg ccgagttcca cgatcaggtg cccttcgatg gcatgtggat cgacatgaac     1560

gagcccagca acttcatcag gggcagcgag gatggctgcc ccaacaacga actggaaaat     1620

cctccttacg tgccaggcgt tgtcggagga acactgcagg ccgccacaat ttgtgccagc     1680

agccatcagt ttctgagcac ccactacaac ctgcacaacc tgtacggcct gaccgaggcc     1740

attgcctctc atagagccct ggttaaggcc agaggcaccc ggccttttgt gatcagcaga     1800

agcacatttg ccggccacgg cagatatgcc ggacattgga caggggacgt ttggtctagt     1860

tgggagcagc tggcctctag cgtgcccgag atcctgcagt ttaatctgct gggagtgccc     1920

ctcgtgggag ccgatgtttg tggatttctg ggcaacacct ccgaggaact gtgcgtcaga     1980

tggacacagc tgggcgcctt ctatcccttc atgagaaacc acaacagcct gctgagcctg     2040

cctcaagagc cttacagctt tagcgaaccc gcacagcagg ccatgagaaa ggccctgact     2100

ctgagatacg ctctgctgcc ccacctgtac accctgtttc atcaagctca tgtggccggc     2160

gagacagtgg ccagaccact gtttctggaa ttccccaagg acagcagcac ctggacagtg     2220

gatcatcagc tgctctgggg agaagccctg ctcattacac ctgtgctgca ggctggcaag     2280

gccgaagtga caggatactt tcccctcggc acttggtacg acctgcagac agttcctgtg     2340

gaagctctgg gatctctgcc tccacctcct gctgctccta gagagcctgc cattcactct     2400

gaaggccagt gggttacact gcccgctcca ctggacacca tcaatgtgca cctgagagcc     2460

ggctacatca tccctctgca aggccctgga ctgaccacaa ccgaaagcag acagcagcca     2520

atggctctgg ccgtggctct gacaaaaggc ggagaagcta gaggcgaact gttctgggat     2580

gacggcgaga gcctggaagt gctggaacgg ggagcctaca cacaagtgat ctttctcgcc     2640

cggaacaaca ccatcgtgaa cgaactcgtc agagtgacca gtgaaggtgc cggactgcag     2700

ctccagaaag tgacagtgct tggagtggcc acagcacccc agcaggtttt gtctaatggc     2760

gtgcccgtgt ccaacttcac atacagccct gacaccaagg tgctggacat ctgtgtgtct     2820

ctgctgatgg gcgagcagtt cctggtgtcc tggtgttga                            2859


<210>  7
<211>  92
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  MVM intron

<400>  7
aagaggtaag ggtttaaggg atggttggtt ggtggggtat taatgtttaa ttacctggag       60

cacctgcctg aaatcacttt ttttcaggtt gg                                     92


<210>  8
<211>  49
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  synthetic Polyadenylation signal

<400>  8
aataaaagat ctttattttc attagatctg tgtgttggtt ttttgtgtg                   49


<210>  9
<211>  7173
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  plasmid AAVss-Dph-CRE02-CSkSH1-SPc5-12-MVM-hGAAco-SynthpA

<400>  9
acatgtcctg caggcagctg cgcgctcgct cgctcactga ggccgcccgg gcaaagcccg       60

ggcgtcgggc gacctttggt cgcccggcct cagtgagcga gcgagcgcgc agagagggag      120

tggccaactc catcactagg ggttcctgcg gccgcacgcg taccggtgac aggtgcggtt      180

cccggagcgc aggcgcacac atgcacccac cggcgaacgc ggtgaccctc gccccacccc      240

atcccctccg gcgggcaact gggtcgggtc aggaggggca aacccgctag ggagacactc      300

catatacggc ccggcccgcg ttacctggga ccgggccaac ccgctccttc tttggtcaac      360

gcaggggacc cgggcggggg cccaggccgc gaaccggccg agggaggggg ctctagtgcc      420

caacacccaa atatggctcg agaagggcag cgacattcct gcggggtggc gcggagggaa      480

tgcccgcggg ctatataaaa cctgagcaga gggacaagcg gccaccgcag cggacagcgc      540

caagtgaagc ctcgcttccc ctccgcggcg accagggccc gagccgagag tagcagttgt      600

agctacccgc ccaggtaggg gcgcgccacg cgtgttctcc tctataaata cccgctctgg      660

tatttggggt tggcagctgt tgctgccagg gagatggttg ggttgacatg cggctcctga      720

caaaacacaa acccctggtg tgtgtgggcg tgggtggtgt gagtaggggg atgaatcagg      780

gagggggcgg gggacccagg gggcaggagc cacacaaagt ctgtgcgggg gtgggagcgc      840

acatagcaat tggaaactga aagcttatca gaccctttct ggaaatcagc ccactgttta      900

taaacttgag gccccaccct cgacagtacc ggggaggaag agggcctgca ctagtccaga      960

gggaaactga ggctcagggc tagctcgccc atagacatac atggcaggca ggctggtacc     1020

caacccgtta cgtggccacc gccttcggca ccatcctcac gacacccaaa tatggcgacg     1080

ggtgaggaat ggtggggagt tatttttaga gcggtgagga aggtgggcag gcagcaggtg     1140

ttggcgctct aaaaataact cccgggagtt atttttagag cggaggaatg gtggacaccc     1200

aaatatggcg acggttcctc acccgtcgcc atatttgggt gtccgccctc ggccggggcc     1260

gcattcctgg gggccgggcg gtgctcccgc ccgcctcgat aaaaggctcc ggggccggcg     1320

gcggcccacg agctacccgg aggagcggga ggcgccaagc tctagatcta gaactagtaa     1380

gaggtaaggg tttaagggat ggttggttgg tggggtatta atgtttaatt acctggagca     1440

cctgcctgaa atcacttttt ttcaggttgg cgtacggcca ccatgggcgt cagacatcct     1500

ccatgttctc acagactgct ggccgtgtgt gctctggtgt ctcttgctac agctgccctg     1560

ctgggacata tcctgctgca cgattttctg ctggtgccca gagagctgtc tggcagctct     1620

cctgtgctgg aagaaacaca ccctgcacat cagcagggcg cctctagacc tggacctaga     1680

gatgctcaag cccatcctgg cagacctaga gccgtgccta cacagtgtga cgtgccacct     1740

aacagcagat tcgactgcgc ccctgacaag gccatcacac aagagcagtg tgaagccaga     1800

ggctgctgct acattcctgc caaacaagga ctgcagggcg ctcagatggg acagccttgg     1860

tgcttcttcc caccatctta ccccagctac aagctggaaa acctgagcag cagcgagatg     1920

ggctacaccg ccacactgac cagaaccaca cctacattct tcccaaagga catcctgaca     1980

ctgcggctgg acgtgatgat ggaaaccgag aaccggctgc acttcaccat caaggacccc     2040

gccaatagaa gatacgaggt gcccctggaa acccctcacg tgcactctag agccccatct     2100

ccactgtaca gcgtggaatt cagcgaggaa ccctttggcg tgatcgtgcg gagacagctg     2160

gatggcagag tgctgctgaa taccacagtg gcccctctgt tcttcgccga ccagtttctg     2220

cagctgagca caagcctgcc tagccagtat atcacaggcc tggccgaaca cctgtctcca     2280

ctgatgctga gcaccagctg gaccagaatc accctgtgga acagagatct ggcccctaca     2340

cctggcgcca atctgtacgg ctctcaccct ttttatctgg ccctggaaga tggcggaagc     2400

gcccacggtg tctttctgct gaacagcaac gccatggacg tggtgctgca accatctcct     2460

gctctgtctt ggagaagcac cggcggcatc ctggacgtgt acatctttct gggacccgag     2520

cctaagagcg tggtgcagca gtatctggat gtcgtgggct accccttcat gcctccttat     2580

tggggcctgg gcttccacct gtgtagatgg ggatacagct ccaccgccat caccagacag     2640

gtggtggaaa acatgacccg ggctcacttc ccactggatg tgcagtggaa cgacctggac     2700

tacatggact ccagacggga cttcaccttt aacaaggacg gcttcagaga cttccccgcc     2760

atggtgcaag aactgcatca aggcggcaga cggtacatga tgatcgtgga tcctgccatc     2820

tcttctagcg gccctgccgg aagctacaga ccttatgatg agggcctgag aagaggcgtg     2880

ttcatcacca atgagacagg ccagcctctg atcggcaaag tgtggcctgg aagcaccgcc     2940

tttccagact tcaccaatcc aaccgctctg gcttggtggg aagatatggt ggccgagttc     3000

cacgatcagg tgcccttcga tggcatgtgg atcgacatga acgagcccag caacttcatc     3060

aggggcagcg aggatggctg ccccaacaac gaactggaaa atcctcctta cgtgccaggc     3120

gttgtcggag gaacactgca ggccgccaca atttgtgcca gcagccatca gtttctgagc     3180

acccactaca acctgcacaa cctgtacggc ctgaccgagg ccattgcctc tcatagagcc     3240

ctggttaagg ccagaggcac ccggcctttt gtgatcagca gaagcacatt tgccggccac     3300

ggcagatatg ccggacattg gacaggggac gtttggtcta gttgggagca gctggcctct     3360

agcgtgcccg agatcctgca gtttaatctg ctgggagtgc ccctcgtggg agccgatgtt     3420

tgtggatttc tgggcaacac ctccgaggaa ctgtgcgtca gatggacaca gctgggcgcc     3480

ttctatccct tcatgagaaa ccacaacagc ctgctgagcc tgcctcaaga gccttacagc     3540

tttagcgaac ccgcacagca ggccatgaga aaggccctga ctctgagata cgctctgctg     3600

ccccacctgt acaccctgtt tcatcaagct catgtggccg gcgagacagt ggccagacca     3660

ctgtttctgg aattccccaa ggacagcagc acctggacag tggatcatca gctgctctgg     3720

ggagaagccc tgctcattac acctgtgctg caggctggca aggccgaagt gacaggatac     3780

tttcccctcg gcacttggta cgacctgcag acagttcctg tggaagctct gggatctctg     3840

cctccacctc ctgctgctcc tagagagcct gccattcact ctgaaggcca gtgggttaca     3900

ctgcccgctc cactggacac catcaatgtg cacctgagag ccggctacat catccctctg     3960

caaggccctg gactgaccac aaccgaaagc agacagcagc caatggctct ggccgtggct     4020

ctgacaaaag gcggagaagc tagaggcgaa ctgttctggg atgacggcga gagcctggaa     4080

gtgctggaac ggggagccta cacacaagtg atctttctcg cccggaacaa caccatcgtg     4140

aacgaactcg tcagagtgac cagtgaaggt gccggactgc agctccagaa agtgacagtg     4200

cttggagtgg ccacagcacc ccagcaggtt ttgtctaatg gcgtgcccgt gtccaacttc     4260

acatacagcc ctgacaccaa ggtgctggac atctgtgtgt ctctgctgat gggcgagcag     4320

ttcctggtgt cctggtgttg acctaggaat aaaagatctt tattttcatt agatctgtgt     4380

gttggttttt tgtgtgaccc gtctgatttt gtaggtaacc acgtgcggac cgagcggccg     4440

caggaacccc tagtgatgga gttggccact ccctctctgc gcgctcgctc gctcactgag     4500

gccgggcgac caaaggtcgc ccgacgcccg ggctttgccc gggcggcctc agtgagcgag     4560

cgagcgcgca gctgcctgca ggggcgcctg atgcggtatt ttctccttac gcatctgtgc     4620

ggtatttcac accgcatacg tcaaagcaac catagtacgc gccctgtagc ggcgcattaa     4680

gcgcggcggg tgtggtggtt acgcgcagcg tgaccgctac acttgccagc gccctagcgc     4740

ccgctccttt cgctttcttc ccttcctttc tcgccacgtt cgccggcttt ccccgtcaag     4800

ctctaaatcg ggggctccct ttagggttcc gatttagtgc tttacggcac ctcgacccca     4860

aaaaacttga tttgggtgat ggttcacgta gtgggccatc gccctgatag acggtttttc     4920

gccctttgac gttggagtcc acgttcttta atagtggact cttgttccaa actggaacaa     4980

cactcaaccc tatctcgggc tattcttttg atttataagg gattttgccg atttcggcct     5040

attggttaaa aaatgagctg atttaacaaa aatttaacgc gaattttaac aaaatattaa     5100

cgtttacaat tttatggtgc actctcagta caatctgctc tgatgccgca tagttaagcc     5160

agccccgaca cccgccaaca cccgctgacg cgccctgacg ggcttgtctg ctcccggcat     5220

ccgcttacag acaagctgtg accgtctccg ggagctgcat gtgtcagagg ttttcaccgt     5280

catcaccgaa acgcgcgaga cgaaagggcc tcgtgatacg cctattttta taggttaatg     5340

tcatgataat aatggtttct tagacgtcag gtggcacttt tcggggaaat gtgcgcggaa     5400

cccctatttg tttatttttc taaatacatt caaatatgta tccgctcatg agacaataac     5460

cctgataaat gcttcaataa tattgaaaaa ggaagagtat gagtattcaa catttccgtg     5520

tcgcccttat tccctttttt gcggcatttt gccttcctgt ttttgctcac ccagaaacgc     5580

tggtgaaagt aaaagatgct gaagatcagt tgggtgcacg agtgggttac atcgaactgg     5640

atctcaacag cggtaagatc cttgagagtt ttcgccccga agaacgtttt ccaatgatga     5700

gcacttttaa agttctgcta tgtggcgcgg tattatcccg tattgacgcc gggcaagagc     5760

aactcggtcg ccgcatacac tattctcaga atgacttggt tgagtactca ccagtcacag     5820

aaaagcatct tacggatggc atgacagtaa gagaattatg cagtgctgcc ataaccatga     5880

gtgataacac tgcggccaac ttacttctga caacgatcgg aggaccgaag gagctaaccg     5940

cttttttgca caacatgggg gatcatgtaa ctcgccttga tcgttgggaa ccggagctga     6000

atgaagccat accaaacgac gagcgtgaca ccacgatgcc tgtagcaatg gcaacaacgt     6060

tgcgcaaact attaactggc gaactactta ctctagcttc ccggcaacaa ttaatagact     6120

ggatggaggc ggataaagtt gcaggaccac ttctgcgctc ggcccttccg gctggctggt     6180

ttattgctga taaatctgga gccggtgagc gtgggtctcg cggtatcatt gcagcactgg     6240

ggccagatgg taagccctcc cgtatcgtag ttatctacac gacggggagt caggcaacta     6300

tggatgaacg aaatagacag atcgctgaga taggtgcctc actgattaag cattggtaac     6360

tgtcagacca agtttactca tatatacttt agattgattt aaaacttcat ttttaattta     6420

aaaggatcta ggtgaagatc ctttttgata atctcatgac caaaatccct taacgtgagt     6480

tttcgttcca ctgagcgtca gaccccgtag aaaagatcaa aggatcttct tgagatcctt     6540

tttttctgcg cgtaatctgc tgcttgcaaa caaaaaaacc accgctacca gcggtggttt     6600

gtttgccgga tcaagagcta ccaactcttt ttccgaaggt aactggcttc agcagagcgc     6660

agataccaaa tactgtcctt ctagtgtagc cgtagttagg ccaccacttc aagaactctg     6720

tagcaccgcc tacatacctc gctctgctaa tcctgttacc agtggctgct gccagtggcg     6780

ataagtcgtg tcttaccggg ttggactcaa gacgatagtt accggataag gcgcagcggt     6840

cgggctgaac ggggggttcg tgcacacagc ccagcttgga gcgaacgacc tacaccgaac     6900

tgagatacct acagcgtgag ctatgagaaa gcgccacgct tcccgaaggg agaaaggcgg     6960

acaggtatcc ggtaagcggc agggtcggaa caggagagcg cacgagggag cttccagggg     7020

gaaacgcctg gtatctttat agtcctgtcg ggtttcgcca cctctgactt gagcgtcgat     7080

ttttgtgatg ctcgtcaggg gggcggagcc tatggaaaaa cgccagcaac gcggcctttt     7140

tacggttcct ggccttttgc tggccttttg ctc                                  7173


<210>  10
<211>  6324
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  plasmid AAVss-SPc5-12-MVM-hGAAco-SynthpA

<400>  10
acatgtcctg caggcagctg cgcgctcgct cgctcactga ggccgcccgg gcaaagcccg       60

ggcgtcgggc gacctttggt cgcccggcct cagtgagcga gcgagcgcgc agagagggag      120

tggccaactc catcactagg ggttcctgcg gccgcacgcg taccgggtac ccaacccgtt      180

acgtggccac cgccttcggc accatcctca cgacacccaa atatggcgac gggtgaggaa      240

tggtggggag ttatttttag agcggtgagg aaggtgggca ggcagcaggt gttggcgctc      300

taaaaataac tcccgggagt tatttttaga gcggaggaat ggtggacacc caaatatggc      360

gacggttcct cacccgtcgc catatttggg tgtccgccct cggccggggc cgcattcctg      420

ggggccgggc ggtgctcccg cccgcctcga taaaaggctc cggggccggc ggcggcccac      480

gagctacccg gaggagcggg aggcgccaag ctctagatct agaactagta agaggtaagg      540

gtttaaggga tggttggttg gtggggtatt aatgtttaat tacctggagc acctgcctga      600

aatcactttt tttcaggttg gcgtacggcc accatgggcg tcagacatcc tccatgttct      660

cacagactgc tggccgtgtg tgctctggtg tctcttgcta cagctgccct gctgggacat      720

atcctgctgc acgattttct gctggtgccc agagagctgt ctggcagctc tcctgtgctg      780

gaagaaacac accctgcaca tcagcagggc gcctctagac ctggacctag agatgctcaa      840

gcccatcctg gcagacctag agccgtgcct acacagtgtg acgtgccacc taacagcaga      900

ttcgactgcg cccctgacaa ggccatcaca caagagcagt gtgaagccag aggctgctgc      960

tacattcctg ccaaacaagg actgcagggc gctcagatgg gacagccttg gtgcttcttc     1020

ccaccatctt accccagcta caagctggaa aacctgagca gcagcgagat gggctacacc     1080

gccacactga ccagaaccac acctacattc ttcccaaagg acatcctgac actgcggctg     1140

gacgtgatga tggaaaccga gaaccggctg cacttcacca tcaaggaccc cgccaataga     1200

agatacgagg tgcccctgga aacccctcac gtgcactcta gagccccatc tccactgtac     1260

agcgtggaat tcagcgagga accctttggc gtgatcgtgc ggagacagct ggatggcaga     1320

gtgctgctga ataccacagt ggcccctctg ttcttcgccg accagtttct gcagctgagc     1380

acaagcctgc ctagccagta tatcacaggc ctggccgaac acctgtctcc actgatgctg     1440

agcaccagct ggaccagaat caccctgtgg aacagagatc tggcccctac acctggcgcc     1500

aatctgtacg gctctcaccc tttttatctg gccctggaag atggcggaag cgcccacggt     1560

gtctttctgc tgaacagcaa cgccatggac gtggtgctgc aaccatctcc tgctctgtct     1620

tggagaagca ccggcggcat cctggacgtg tacatctttc tgggacccga gcctaagagc     1680

gtggtgcagc agtatctgga tgtcgtgggc taccccttca tgcctcctta ttggggcctg     1740

ggcttccacc tgtgtagatg gggatacagc tccaccgcca tcaccagaca ggtggtggaa     1800

aacatgaccc gggctcactt cccactggat gtgcagtgga acgacctgga ctacatggac     1860

tccagacggg acttcacctt taacaaggac ggcttcagag acttccccgc catggtgcaa     1920

gaactgcatc aaggcggcag acggtacatg atgatcgtgg atcctgccat ctcttctagc     1980

ggccctgccg gaagctacag accttatgat gagggcctga gaagaggcgt gttcatcacc     2040

aatgagacag gccagcctct gatcggcaaa gtgtggcctg gaagcaccgc ctttccagac     2100

ttcaccaatc caaccgctct ggcttggtgg gaagatatgg tggccgagtt ccacgatcag     2160

gtgcccttcg atggcatgtg gatcgacatg aacgagccca gcaacttcat caggggcagc     2220

gaggatggct gccccaacaa cgaactggaa aatcctcctt acgtgccagg cgttgtcgga     2280

ggaacactgc aggccgccac aatttgtgcc agcagccatc agtttctgag cacccactac     2340

aacctgcaca acctgtacgg cctgaccgag gccattgcct ctcatagagc cctggttaag     2400

gccagaggca cccggccttt tgtgatcagc agaagcacat ttgccggcca cggcagatat     2460

gccggacatt ggacagggga cgtttggtct agttgggagc agctggcctc tagcgtgccc     2520

gagatcctgc agtttaatct gctgggagtg cccctcgtgg gagccgatgt ttgtggattt     2580

ctgggcaaca cctccgagga actgtgcgtc agatggacac agctgggcgc cttctatccc     2640

ttcatgagaa accacaacag cctgctgagc ctgcctcaag agccttacag ctttagcgaa     2700

cccgcacagc aggccatgag aaaggccctg actctgagat acgctctgct gccccacctg     2760

tacaccctgt ttcatcaagc tcatgtggcc ggcgagacag tggccagacc actgtttctg     2820

gaattcccca aggacagcag cacctggaca gtggatcatc agctgctctg gggagaagcc     2880

ctgctcatta cacctgtgct gcaggctggc aaggccgaag tgacaggata ctttcccctc     2940

ggcacttggt acgacctgca gacagttcct gtggaagctc tgggatctct gcctccacct     3000

cctgctgctc ctagagagcc tgccattcac tctgaaggcc agtgggttac actgcccgct     3060

ccactggaca ccatcaatgt gcacctgaga gccggctaca tcatccctct gcaaggccct     3120

ggactgacca caaccgaaag cagacagcag ccaatggctc tggccgtggc tctgacaaaa     3180

ggcggagaag ctagaggcga actgttctgg gatgacggcg agagcctgga agtgctggaa     3240

cggggagcct acacacaagt gatctttctc gcccggaaca acaccatcgt gaacgaactc     3300

gtcagagtga ccagtgaagg tgccggactg cagctccaga aagtgacagt gcttggagtg     3360

gccacagcac cccagcaggt tttgtctaat ggcgtgcccg tgtccaactt cacatacagc     3420

cctgacacca aggtgctgga catctgtgtg tctctgctga tgggcgagca gttcctggtg     3480

tcctggtgtt gacctaggaa taaaagatct ttattttcat tagatctgtg tgttggtttt     3540

ttgtgtgacc cgtctgattt tgtaggtaac cacgtgcgga ccgagcggcc gcaggaaccc     3600

ctagtgatgg agttggccac tccctctctg cgcgctcgct cgctcactga ggccgggcga     3660

ccaaaggtcg cccgacgccc gggctttgcc cgggcggcct cagtgagcga gcgagcgcgc     3720

agctgcctgc aggggcgcct gatgcggtat tttctcctta cgcatctgtg cggtatttca     3780

caccgcatac gtcaaagcaa ccatagtacg cgccctgtag cggcgcatta agcgcggcgg     3840

gtgtggtggt tacgcgcagc gtgaccgcta cacttgccag cgccctagcg cccgctcctt     3900

tcgctttctt cccttccttt ctcgccacgt tcgccggctt tccccgtcaa gctctaaatc     3960

gggggctccc tttagggttc cgatttagtg ctttacggca cctcgacccc aaaaaacttg     4020

atttgggtga tggttcacgt agtgggccat cgccctgata gacggttttt cgccctttga     4080

cgttggagtc cacgttcttt aatagtggac tcttgttcca aactggaaca acactcaacc     4140

ctatctcggg ctattctttt gatttataag ggattttgcc gatttcggcc tattggttaa     4200

aaaatgagct gatttaacaa aaatttaacg cgaattttaa caaaatatta acgtttacaa     4260

ttttatggtg cactctcagt acaatctgct ctgatgccgc atagttaagc cagccccgac     4320

acccgccaac acccgctgac gcgccctgac gggcttgtct gctcccggca tccgcttaca     4380

gacaagctgt gaccgtctcc gggagctgca tgtgtcagag gttttcaccg tcatcaccga     4440

aacgcgcgag acgaaagggc ctcgtgatac gcctattttt ataggttaat gtcatgataa     4500

taatggtttc ttagacgtca ggtggcactt ttcggggaaa tgtgcgcgga acccctattt     4560

gtttattttt ctaaatacat tcaaatatgt atccgctcat gagacaataa ccctgataaa     4620

tgcttcaata atattgaaaa aggaagagta tgagtattca acatttccgt gtcgccctta     4680

ttcccttttt tgcggcattt tgccttcctg tttttgctca cccagaaacg ctggtgaaag     4740

taaaagatgc tgaagatcag ttgggtgcac gagtgggtta catcgaactg gatctcaaca     4800

gcggtaagat ccttgagagt tttcgccccg aagaacgttt tccaatgatg agcactttta     4860

aagttctgct atgtggcgcg gtattatccc gtattgacgc cgggcaagag caactcggtc     4920

gccgcataca ctattctcag aatgacttgg ttgagtactc accagtcaca gaaaagcatc     4980

ttacggatgg catgacagta agagaattat gcagtgctgc cataaccatg agtgataaca     5040

ctgcggccaa cttacttctg acaacgatcg gaggaccgaa ggagctaacc gcttttttgc     5100

acaacatggg ggatcatgta actcgccttg atcgttggga accggagctg aatgaagcca     5160

taccaaacga cgagcgtgac accacgatgc ctgtagcaat ggcaacaacg ttgcgcaaac     5220

tattaactgg cgaactactt actctagctt cccggcaaca attaatagac tggatggagg     5280

cggataaagt tgcaggacca cttctgcgct cggcccttcc ggctggctgg tttattgctg     5340

ataaatctgg agccggtgag cgtgggtctc gcggtatcat tgcagcactg gggccagatg     5400

gtaagccctc ccgtatcgta gttatctaca cgacggggag tcaggcaact atggatgaac     5460

gaaatagaca gatcgctgag ataggtgcct cactgattaa gcattggtaa ctgtcagacc     5520

aagtttactc atatatactt tagattgatt taaaacttca tttttaattt aaaaggatct     5580

aggtgaagat cctttttgat aatctcatga ccaaaatccc ttaacgtgag ttttcgttcc     5640

actgagcgtc agaccccgta gaaaagatca aaggatcttc ttgagatcct ttttttctgc     5700

gcgtaatctg ctgcttgcaa acaaaaaaac caccgctacc agcggtggtt tgtttgccgg     5760

atcaagagct accaactctt tttccgaagg taactggctt cagcagagcg cagataccaa     5820

atactgtcct tctagtgtag ccgtagttag gccaccactt caagaactct gtagcaccgc     5880

ctacatacct cgctctgcta atcctgttac cagtggctgc tgccagtggc gataagtcgt     5940

gtcttaccgg gttggactca agacgatagt taccggataa ggcgcagcgg tcgggctgaa     6000

cggggggttc gtgcacacag cccagcttgg agcgaacgac ctacaccgaa ctgagatacc     6060

tacagcgtga gctatgagaa agcgccacgc ttcccgaagg gagaaaggcg gacaggtatc     6120

cggtaagcgg cagggtcgga acaggagagc gcacgaggga gcttccaggg ggaaacgcct     6180

ggtatcttta tagtcctgtc gggtttcgcc acctctgact tgagcgtcga tttttgtgat     6240

gctcgtcagg ggggcggagc ctatggaaaa acgccagcaa cgcggccttt ttacggttcc     6300

tggccttttg ctggcctttt gctc                                            6324


<210>  11
<211>  7173
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  plasmid AAVss-Dph-CRE02-CSkSH1-SPc5-12-MVM-hGAAwt-SynthpA

<400>  11
acatgtcctg caggcagctg cgcgctcgct cgctcactga ggccgcccgg gcaaagcccg       60

ggcgtcgggc gacctttggt cgcccggcct cagtgagcga gcgagcgcgc agagagggag      120

tggccaactc catcactagg ggttcctgcg gccgcacgcg taccggtgac aggtgcggtt      180

cccggagcgc aggcgcacac atgcacccac cggcgaacgc ggtgaccctc gccccacccc      240

atcccctccg gcgggcaact gggtcgggtc aggaggggca aacccgctag ggagacactc      300

catatacggc ccggcccgcg ttacctggga ccgggccaac ccgctccttc tttggtcaac      360

gcaggggacc cgggcggggg cccaggccgc gaaccggccg agggaggggg ctctagtgcc      420

caacacccaa atatggctcg agaagggcag cgacattcct gcggggtggc gcggagggaa      480

tgcccgcggg ctatataaaa cctgagcaga gggacaagcg gccaccgcag cggacagcgc      540

caagtgaagc ctcgcttccc ctccgcggcg accagggccc gagccgagag tagcagttgt      600

agctacccgc ccaggtaggg gcgcgccacg cgtgttctcc tctataaata cccgctctgg      660

tatttggggt tggcagctgt tgctgccagg gagatggttg ggttgacatg cggctcctga      720

caaaacacaa acccctggtg tgtgtgggcg tgggtggtgt gagtaggggg atgaatcagg      780

gagggggcgg gggacccagg gggcaggagc cacacaaagt ctgtgcgggg gtgggagcgc      840

acatagcaat tggaaactga aagcttatca gaccctttct ggaaatcagc ccactgttta      900

taaacttgag gccccaccct cgacagtacc ggggaggaag agggcctgca ctagtccaga      960

gggaaactga ggctcagggc tagctcgccc atagacatac atggcaggca ggctggtacc     1020

caacccgtta cgtggccacc gccttcggca ccatcctcac gacacccaaa tatggcgacg     1080

ggtgaggaat ggtggggagt tatttttaga gcggtgagga aggtgggcag gcagcaggtg     1140

ttggcgctct aaaaataact cccgggagtt atttttagag cggaggaatg gtggacaccc     1200

aaatatggcg acggttcctc acccgtcgcc atatttgggt gtccgccctc ggccggggcc     1260

gcattcctgg gggccgggcg gtgctcccgc ccgcctcgat aaaaggctcc ggggccggcg     1320

gcggcccacg agctacccgg aggagcggga ggcgccaagc tctagatcta gaactagtaa     1380

gaggtaaggg tttaagggat ggttggttgg tggggtatta atgtttaatt acctggagca     1440

cctgcctgaa atcacttttt ttcaggttgg cgtacggcca ccatgggagt gaggcacccg     1500

ccctgctccc accggctcct ggccgtctgc gccctcgtgt ccttggcaac cgctgcactc     1560

ctggggcaca tcctactcca tgatttcctg ctggttcccc gagagctgag tggctcctcc     1620

ccagtcctgg aggagactca cccagctcac cagcagggag ccagcagacc agggccccgg     1680

gatgcccagg cacaccccgg ccgtcccaga gcagtgccca cacagtgcga cgtccccccc     1740

aacagccgct tcgattgcgc ccctgacaag gccatcaccc aggaacagtg cgaggcccgc     1800

ggctgttgct acatccctgc aaagcagggg ctgcagggag cccagatggg gcagccctgg     1860

tgcttcttcc cacccagcta ccccagctac aagctggaga acctgagctc ctctgaaatg     1920

ggctacacgg ccaccctgac ccgtaccacc cccaccttct tccccaagga catcctgacc     1980

ctgcggctgg acgtgatgat ggagactgag aaccgcctcc acttcacgat caaagatcca     2040

gctaacaggc gctacgaggt gcccttggag accccgcatg tccacagccg ggcaccgtcc     2100

ccactctaca gcgtggagtt ctccgaggag cccttcgggg tgatcgtgcg ccggcagctg     2160

gacggccgcg tgctgctgaa cacgacggtg gcgcccctgt tctttgcgga ccagttcctt     2220

cagctgtcca cctcgctgcc ctcgcagtat atcacaggcc tcgccgagca cctcagtccc     2280

ctgatgctca gcaccagctg gaccaggatc accctgtgga accgggacct tgcgcccacg     2340

cccggtgcga acctctacgg gtctcaccct ttctacctgg cgctggagga cggcgggtcg     2400

gcacacgggg tgttcctgct aaacagcaat gccatggatg tggtcctgca gccgagccct     2460

gcccttagct ggaggtcgac aggtgggatc ctggatgtct acatcttcct gggcccagag     2520

cccaagagcg tggtgcagca gtacctggac gttgtgggat acccgttcat gccgccatac     2580

tggggcctgg gcttccacct gtgccgctgg ggctactcct ccaccgctat cacccgccag     2640

gtggtggaga acatgaccag ggcccacttc cccctggacg tccagtggaa cgacctggac     2700

tacatggact cccggaggga cttcacgttc aacaaggatg gcttccggga cttcccggcc     2760

atggtgcagg agctgcacca gggcggccgg cgctacatga tgatcgtgga tcctgccatc     2820

agcagctcgg gccctgccgg gagctacagg ccctacgacg agggtctgcg gaggggggtt     2880

ttcatcacca acgagaccgg ccagccgctg attgggaagg tatggcccgg gtccactgcc     2940

ttccccgact tcaccaaccc cacagccctg gcctggtggg aggacatggt ggctgagttc     3000

catgaccagg tgcccttcga cggcatgtgg attgacatga acgagccttc caacttcatc     3060

aggggctctg aggacggctg ccccaacaat gagctggaga acccacccta cgtgcctggg     3120

gtggttgggg ggaccctcca ggcggccacc atctgtgcct ccagccacca gtttctctcc     3180

acacactaca acctgcacaa cctctacggc ctgaccgaag ccatcgcctc ccacagggcg     3240

ctggtgaagg ctcgggggac acgcccattt gtgatctccc gctcgacctt tgctggccac     3300

ggccgatacg ccggccactg gacgggggac gtgtggagct cctgggagca gctcgcctcc     3360

tccgtgccag aaatcctgca gtttaacctg ctgggggtgc ctctggtcgg ggccgacgtc     3420

tgcggcttcc tgggcaacac ctcagaggag ctgtgtgtgc gctggaccca gctgggggcc     3480

ttctacccct tcatgcggaa ccacaacagc ctgctcagtc tgccccagga gccgtacagc     3540

ttcagcgagc cggcccagca ggccatgagg aaggccctca ccctgcgcta cgcactcctc     3600

ccccacctct acacactgtt ccaccaggcc cacgtcgcgg gggagaccgt ggcccggccc     3660

ctcttcctgg agttccccaa ggactctagc acctggactg tggaccacca gctcctgtgg     3720

ggggaggccc tgctcatcac cccagtgctc caggccggga aggccgaagt gactggctac     3780

ttccccttgg gcacatggta cgacctgcag acggtgccag tagaggccct tggcagcctc     3840

ccacccccac ctgcagctcc ccgtgagcca gccatccaca gcgaggggca gtgggtgacg     3900

ctgccggccc ccctggacac catcaacgtc cacctccggg ctgggtacat catccccctg     3960

cagggccctg gcctcacaac cacagagtcc cgccagcagc ccatggccct ggctgtggcc     4020

ctgaccaagg gtggggaggc ccgaggggag ctgttctggg acgatggaga gagcctggaa     4080

gtgctggagc gaggggccta cacacaggtc atcttcctgg ccaggaataa cacgatcgtg     4140

aatgagctgg tacgtgtgac cagtgaggga gctggcctgc agctgcagaa ggtgactgtc     4200

ctgggcgtgg ccacggcgcc ccagcaggtc ctctccaacg gtgtccctgt ctccaacttc     4260

acctacagcc ccgacaccaa ggtcctggac atctgtgtct cgctgttgat gggagagcag     4320

tttctcgtca gctggtgtta gcctaggaat aaaagatctt tattttcatt agatctgtgt     4380

gttggttttt tgtgtgaccc gtctgatttt gtaggtaacc acgtgcggac cgagcggccg     4440

caggaacccc tagtgatgga gttggccact ccctctctgc gcgctcgctc gctcactgag     4500

gccgggcgac caaaggtcgc ccgacgcccg ggctttgccc gggcggcctc agtgagcgag     4560

cgagcgcgca gctgcctgca ggggcgcctg atgcggtatt ttctccttac gcatctgtgc     4620

ggtatttcac accgcatacg tcaaagcaac catagtacgc gccctgtagc ggcgcattaa     4680

gcgcggcggg tgtggtggtt acgcgcagcg tgaccgctac acttgccagc gccctagcgc     4740

ccgctccttt cgctttcttc ccttcctttc tcgccacgtt cgccggcttt ccccgtcaag     4800

ctctaaatcg ggggctccct ttagggttcc gatttagtgc tttacggcac ctcgacccca     4860

aaaaacttga tttgggtgat ggttcacgta gtgggccatc gccctgatag acggtttttc     4920

gccctttgac gttggagtcc acgttcttta atagtggact cttgttccaa actggaacaa     4980

cactcaaccc tatctcgggc tattcttttg atttataagg gattttgccg atttcggcct     5040

attggttaaa aaatgagctg atttaacaaa aatttaacgc gaattttaac aaaatattaa     5100

cgtttacaat tttatggtgc actctcagta caatctgctc tgatgccgca tagttaagcc     5160

agccccgaca cccgccaaca cccgctgacg cgccctgacg ggcttgtctg ctcccggcat     5220

ccgcttacag acaagctgtg accgtctccg ggagctgcat gtgtcagagg ttttcaccgt     5280

catcaccgaa acgcgcgaga cgaaagggcc tcgtgatacg cctattttta taggttaatg     5340

tcatgataat aatggtttct tagacgtcag gtggcacttt tcggggaaat gtgcgcggaa     5400

cccctatttg tttatttttc taaatacatt caaatatgta tccgctcatg agacaataac     5460

cctgataaat gcttcaataa tattgaaaaa ggaagagtat gagtattcaa catttccgtg     5520

tcgcccttat tccctttttt gcggcatttt gccttcctgt ttttgctcac ccagaaacgc     5580

tggtgaaagt aaaagatgct gaagatcagt tgggtgcacg agtgggttac atcgaactgg     5640

atctcaacag cggtaagatc cttgagagtt ttcgccccga agaacgtttt ccaatgatga     5700

gcacttttaa agttctgcta tgtggcgcgg tattatcccg tattgacgcc gggcaagagc     5760

aactcggtcg ccgcatacac tattctcaga atgacttggt tgagtactca ccagtcacag     5820

aaaagcatct tacggatggc atgacagtaa gagaattatg cagtgctgcc ataaccatga     5880

gtgataacac tgcggccaac ttacttctga caacgatcgg aggaccgaag gagctaaccg     5940

cttttttgca caacatgggg gatcatgtaa ctcgccttga tcgttgggaa ccggagctga     6000

atgaagccat accaaacgac gagcgtgaca ccacgatgcc tgtagcaatg gcaacaacgt     6060

tgcgcaaact attaactggc gaactactta ctctagcttc ccggcaacaa ttaatagact     6120

ggatggaggc ggataaagtt gcaggaccac ttctgcgctc ggcccttccg gctggctggt     6180

ttattgctga taaatctgga gccggtgagc gtgggtctcg cggtatcatt gcagcactgg     6240

ggccagatgg taagccctcc cgtatcgtag ttatctacac gacggggagt caggcaacta     6300

tggatgaacg aaatagacag atcgctgaga taggtgcctc actgattaag cattggtaac     6360

tgtcagacca agtttactca tatatacttt agattgattt aaaacttcat ttttaattta     6420

aaaggatcta ggtgaagatc ctttttgata atctcatgac caaaatccct taacgtgagt     6480

tttcgttcca ctgagcgtca gaccccgtag aaaagatcaa aggatcttct tgagatcctt     6540

tttttctgcg cgtaatctgc tgcttgcaaa caaaaaaacc accgctacca gcggtggttt     6600

gtttgccgga tcaagagcta ccaactcttt ttccgaaggt aactggcttc agcagagcgc     6660

agataccaaa tactgtcctt ctagtgtagc cgtagttagg ccaccacttc aagaactctg     6720

tagcaccgcc tacatacctc gctctgctaa tcctgttacc agtggctgct gccagtggcg     6780

ataagtcgtg tcttaccggg ttggactcaa gacgatagtt accggataag gcgcagcggt     6840

cgggctgaac ggggggttcg tgcacacagc ccagcttgga gcgaacgacc tacaccgaac     6900

tgagatacct acagcgtgag ctatgagaaa gcgccacgct tcccgaaggg agaaaggcgg     6960

acaggtatcc ggtaagcggc agggtcggaa caggagagcg cacgagggag cttccagggg     7020

gaaacgcctg gtatctttat agtcctgtcg ggtttcgcca cctctgactt gagcgtcgat     7080

ttttgtgatg ctcgtcaggg gggcggagcc tatggaaaaa cgccagcaac gcggcctttt     7140

tacggttcct ggccttttgc tggccttttg ctc                                  7173


<210>  12
<211>  6308
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  plasmid AAVss-SPc5-12-MVM-hGAAwt-SynthpA

<400>  12
acatgtcctg caggcagctg cgcgctcgct cgctcactga ggccgcccgg gcaaagcccg       60

ggcgtcgggc gacctttggt cgcccggcct cagtgagcga gcgagcgcgc agagagggag      120

tggccaactc catcactagg ggttcctgcg gccgcacgcg taccggttgg ccaccgcctt      180

cggcaccatc ctcacgacac ccaaatatgg cgacgggtga ggaatggtgg ggagttattt      240

ttagagcggt gaggaaggtg ggcaggcagc aggtgttggc gctctaaaaa taactcccgg      300

gagttatttt tagagcggag gaatggtgga cacccaaata tggcgacggt tcctcacccg      360

tcgccatatt tgggtgtccg ccctcggccg gggccgcatt cctgggggcc gggcggtgct      420

cccgcccgcc tcgataaaag gctccggggc cggcggcggc ccacgagcta cccggaggag      480

cgggaggcgc caagctctag atctagaact agtaagaggt aagggtttaa gggatggttg      540

gttggtgggg tattaatgtt taattacctg gagcacctgc ctgaaatcac tttttttcag      600

gttggcgtac ggccaccatg ggagtgaggc acccgccctg ctcccaccgg ctcctggccg      660

tctgcgccct cgtgtccttg gcaaccgctg cactcctggg gcacatccta ctccatgatt      720

tcctgctggt tccccgagag ctgagtggct cctccccagt cctggaggag actcacccag      780

ctcaccagca gggagccagc agaccagggc cccgggatgc ccaggcacac cccggccgtc      840

ccagagcagt gcccacacag tgcgacgtcc cccccaacag ccgcttcgat tgcgcccctg      900

acaaggccat cacccaggaa cagtgcgagg cccgcggctg ttgctacatc cctgcaaagc      960

aggggctgca gggagcccag atggggcagc cctggtgctt cttcccaccc agctacccca     1020

gctacaagct ggagaacctg agctcctctg aaatgggcta cacggccacc ctgacccgta     1080

ccacccccac cttcttcccc aaggacatcc tgaccctgcg gctggacgtg atgatggaga     1140

ctgagaaccg cctccacttc acgatcaaag atccagctaa caggcgctac gaggtgccct     1200

tggagacccc gcatgtccac agccgggcac cgtccccact ctacagcgtg gagttctccg     1260

aggagccctt cggggtgatc gtgcgccggc agctggacgg ccgcgtgctg ctgaacacga     1320

cggtggcgcc cctgttcttt gcggaccagt tccttcagct gtccacctcg ctgccctcgc     1380

agtatatcac aggcctcgcc gagcacctca gtcccctgat gctcagcacc agctggacca     1440

ggatcaccct gtggaaccgg gaccttgcgc ccacgcccgg tgcgaacctc tacgggtctc     1500

accctttcta cctggcgctg gaggacggcg ggtcggcaca cggggtgttc ctgctaaaca     1560

gcaatgccat ggatgtggtc ctgcagccga gccctgccct tagctggagg tcgacaggtg     1620

ggatcctgga tgtctacatc ttcctgggcc cagagcccaa gagcgtggtg cagcagtacc     1680

tggacgttgt gggatacccg ttcatgccgc catactgggg cctgggcttc cacctgtgcc     1740

gctggggcta ctcctccacc gctatcaccc gccaggtggt ggagaacatg accagggccc     1800

acttccccct ggacgtccag tggaacgacc tggactacat ggactcccgg agggacttca     1860

cgttcaacaa ggatggcttc cgggacttcc cggccatggt gcaggagctg caccagggcg     1920

gccggcgcta catgatgatc gtggatcctg ccatcagcag ctcgggccct gccgggagct     1980

acaggcccta cgacgagggt ctgcggaggg gggttttcat caccaacgag accggccagc     2040

cgctgattgg gaaggtatgg cccgggtcca ctgccttccc cgacttcacc aaccccacag     2100

ccctggcctg gtgggaggac atggtggctg agttccatga ccaggtgccc ttcgacggca     2160

tgtggattga catgaacgag ccttccaact tcatcagggg ctctgaggac ggctgcccca     2220

acaatgagct ggagaaccca ccctacgtgc ctggggtggt tggggggacc ctccaggcgg     2280

ccaccatctg tgcctccagc caccagtttc tctccacaca ctacaacctg cacaacctct     2340

acggcctgac cgaagccatc gcctcccaca gggcgctggt gaaggctcgg gggacacgcc     2400

catttgtgat ctcccgctcg acctttgctg gccacggccg atacgccggc cactggacgg     2460

gggacgtgtg gagctcctgg gagcagctcg cctcctccgt gccagaaatc ctgcagttta     2520

acctgctggg ggtgcctctg gtcggggccg acgtctgcgg cttcctgggc aacacctcag     2580

aggagctgtg tgtgcgctgg acccagctgg gggccttcta ccccttcatg cggaaccaca     2640

acagcctgct cagtctgccc caggagccgt acagcttcag cgagccggcc cagcaggcca     2700

tgaggaaggc cctcaccctg cgctacgcac tcctccccca cctctacaca ctgttccacc     2760

aggcccacgt cgcgggggag accgtggccc ggcccctctt cctggagttc cccaaggact     2820

ctagcacctg gactgtggac caccagctcc tgtgggggga ggccctgctc atcaccccag     2880

tgctccaggc cgggaaggcc gaagtgactg gctacttccc cttgggcaca tggtacgacc     2940

tgcagacggt gccagtagag gcccttggca gcctcccacc cccacctgca gctccccgtg     3000

agccagccat ccacagcgag gggcagtggg tgacgctgcc ggcccccctg gacaccatca     3060

acgtccacct ccgggctggg tacatcatcc ccctgcaggg ccctggcctc acaaccacag     3120

agtcccgcca gcagcccatg gccctggctg tggccctgac caagggtggg gaggcccgag     3180

gggagctgtt ctgggacgat ggagagagcc tggaagtgct ggagcgaggg gcctacacac     3240

aggtcatctt cctggccagg aataacacga tcgtgaatga gctggtacgt gtgaccagtg     3300

agggagctgg cctgcagctg cagaaggtga ctgtcctggg cgtggccacg gcgccccagc     3360

aggtcctctc caacggtgtc cctgtctcca acttcaccta cagccccgac accaaggtcc     3420

tggacatctg tgtctcgctg ttgatgggag agcagtttct cgtcagctgg tgttagccta     3480

ggaataaaag atctttattt tcattagatc tgtgtgttgg ttttttgtgt gacccgtctg     3540

attttgtagg taaccacgtg cggaccgagc ggccgcagga acccctagtg atggagttgg     3600

ccactccctc tctgcgcgct cgctcgctca ctgaggccgg gcgaccaaag gtcgcccgac     3660

gcccgggctt tgcccgggcg gcctcagtga gcgagcgagc gcgcagctgc ctgcaggggc     3720

gcctgatgcg gtattttctc cttacgcatc tgtgcggtat ttcacaccgc atacgtcaaa     3780

gcaaccatag tacgcgccct gtagcggcgc attaagcgcg gcgggtgtgg tggttacgcg     3840

cagcgtgacc gctacacttg ccagcgccct agcgcccgct cctttcgctt tcttcccttc     3900

ctttctcgcc acgttcgccg gctttccccg tcaagctcta aatcgggggc tccctttagg     3960

gttccgattt agtgctttac ggcacctcga ccccaaaaaa cttgatttgg gtgatggttc     4020

acgtagtggg ccatcgccct gatagacggt ttttcgccct ttgacgttgg agtccacgtt     4080

ctttaatagt ggactcttgt tccaaactgg aacaacactc aaccctatct cgggctattc     4140

ttttgattta taagggattt tgccgatttc ggcctattgg ttaaaaaatg agctgattta     4200

acaaaaattt aacgcgaatt ttaacaaaat attaacgttt acaattttat ggtgcactct     4260

cagtacaatc tgctctgatg ccgcatagtt aagccagccc cgacacccgc caacacccgc     4320

tgacgcgccc tgacgggctt gtctgctccc ggcatccgct tacagacaag ctgtgaccgt     4380

ctccgggagc tgcatgtgtc agaggttttc accgtcatca ccgaaacgcg cgagacgaaa     4440

gggcctcgtg atacgcctat ttttataggt taatgtcatg ataataatgg tttcttagac     4500

gtcaggtggc acttttcggg gaaatgtgcg cggaacccct atttgtttat ttttctaaat     4560

acattcaaat atgtatccgc tcatgagaca ataaccctga taaatgcttc aataatattg     4620

aaaaaggaag agtatgagta ttcaacattt ccgtgtcgcc cttattccct tttttgcggc     4680

attttgcctt cctgtttttg ctcacccaga aacgctggtg aaagtaaaag atgctgaaga     4740

tcagttgggt gcacgagtgg gttacatcga actggatctc aacagcggta agatccttga     4800

gagttttcgc cccgaagaac gttttccaat gatgagcact tttaaagttc tgctatgtgg     4860

cgcggtatta tcccgtattg acgccgggca agagcaactc ggtcgccgca tacactattc     4920

tcagaatgac ttggttgagt actcaccagt cacagaaaag catcttacgg atggcatgac     4980

agtaagagaa ttatgcagtg ctgccataac catgagtgat aacactgcgg ccaacttact     5040

tctgacaacg atcggaggac cgaaggagct aaccgctttt ttgcacaaca tgggggatca     5100

tgtaactcgc cttgatcgtt gggaaccgga gctgaatgaa gccataccaa acgacgagcg     5160

tgacaccacg atgcctgtag caatggcaac aacgttgcgc aaactattaa ctggcgaact     5220

acttactcta gcttcccggc aacaattaat agactggatg gaggcggata aagttgcagg     5280

accacttctg cgctcggccc ttccggctgg ctggtttatt gctgataaat ctggagccgg     5340

tgagcgtggg tctcgcggta tcattgcagc actggggcca gatggtaagc cctcccgtat     5400

cgtagttatc tacacgacgg ggagtcaggc aactatggat gaacgaaata gacagatcgc     5460

tgagataggt gcctcactga ttaagcattg gtaactgtca gaccaagttt actcatatat     5520

actttagatt gatttaaaac ttcattttta atttaaaagg atctaggtga agatcctttt     5580

tgataatctc atgaccaaaa tcccttaacg tgagttttcg ttccactgag cgtcagaccc     5640

cgtagaaaag atcaaaggat cttcttgaga tccttttttt ctgcgcgtaa tctgctgctt     5700

gcaaacaaaa aaaccaccgc taccagcggt ggtttgtttg ccggatcaag agctaccaac     5760

tctttttccg aaggtaactg gcttcagcag agcgcagata ccaaatactg tccttctagt     5820

gtagccgtag ttaggccacc acttcaagaa ctctgtagca ccgcctacat acctcgctct     5880

gctaatcctg ttaccagtgg ctgctgccag tggcgataag tcgtgtctta ccgggttgga     5940

ctcaagacga tagttaccgg ataaggcgca gcggtcgggc tgaacggggg gttcgtgcac     6000

acagcccagc ttggagcgaa cgacctacac cgaactgaga tacctacagc gtgagctatg     6060

agaaagcgcc acgcttcccg aagggagaaa ggcggacagg tatccggtaa gcggcagggt     6120

cggaacagga gagcgcacga gggagcttcc agggggaaac gcctggtatc tttatagtcc     6180

tgtcgggttt cgccacctct gacttgagcg tcgatttttg tgatgctcgt caggggggcg     6240

gagcctatgg aaaaacgcca gcaacgcggc ctttttacgg ttcctggcct tttgctggcc     6300

ttttgctc                                                              6308


<210>  13
<211>  14
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  linker

<400>  13
ggcgcgccac gcgt                                                         14


<210>  14
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  forward primer 1 for AAV vector titration

<400>  14
agggatggtt ggttggtgg                                                    19


<210>  15
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  reverse primer 1 for AAV vector titration

<400>  15
ggcaggtgct ccaggtaat                                                    19


<210>  16
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  forward primer 2 for AAV vector titration

<400>  16
ccatcctcac gacacccaa                                                    19


<210>  17
<211>  18
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  reverse primer 2 for AAV vector titration

<400>  17
gtccaccatt cctccgct                                                     18


<210>  18
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hGAAco forward primer

<400>  18
accccttcat gcctccttat                                                   20


<210>  19
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  hGAAco reverse primer

<400>  19
tccatgtagt ccaggtcgtt                                                   20


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

<220>
<223>  GAPDH forward primer

<400>  20
tgtgtccgtc gtggatctga                                                   20


<210>  21
<211>  22
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  GAPDH reverse primer

<400>  21
gcctgcttca ccaccttctt ga                                                22


<210>  22
<211>  1426
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Desmin promoter

<400>  22
acacacctac tagtaacccc tccagctggt gatggcaggt ctagggtagg accagtgact       60

ggctcctaat cgagcactct attttcaggg tttgcattcc aaaagggtca ggtccaagag      120

ggacctggag tgccaagtgg aggtgtagag gcacggccag tacccatgga gaatggtgga      180

tgtccttagg ggttagcaag tgccgtgtgc taaggagggg gctttggagg ttgggcaggc      240

cctctgtggg gctccatttt tgtgggggtg ggggctggag cattataggg ggtgggaagt      300

gattggggct gtcaccctag ccttccttat ctgacgccca cccatgcctc ctcaggtacc      360

ccctgccccc cacagctcct ctcctgtgcc ttgtttccca gccatgcgtt ctcctctata      420

aatacccgct ctggtatttg gggttggcag ctgttgctgc cagggagatg gttgggttga      480

catgcggctc ctgacaaaac acaaacccct ggtgtgtgtg ggcgtgggtg gtgtgagtag      540

ggggatgaat cagggagggg gcgggggacc cagggggcag gagccacaca aagtctgtgc      600

gggggtggga gcgcacatag caattggaaa ctgaaagctt atcagaccct ttctggaaat      660

cagcccactg tttataaact tgaggcccca ccctcgacag taccggggag gaagagggcc      720

tgcactagtc cagagggaaa ctgaggctca gggctagctc gcccatagac atacatggca      780

ggcaggcttt ggccaggatc cctccgcctg ccaggcgtct ccctgccctc ccttcctgcc      840

tagagacccc caccctcaag cctggctggt ctttgcctga gacccaaacc tcttcgactt      900

caagagaata tttaggaaca aggtggttta gggcctttcc tgggaacagg ccttgaccct      960

ttaagaaatg acccaaagtc tctccttgac caaaaagggg accctcaaac taaagggaag     1020

cctctcttct gctgtctccc ctgaccccac tcccccccac cccaggacga ggagataacc     1080

agggctgaaa gaggcccgcc tgggggctgc agacatgctt gctgcctgcc ctggcgaagg     1140

attggcaggc ttgcccgtca caggaccccc gctggctgac tcaggggcgc aggcctcttg     1200

cgggggagct ggcctccccg cccccacggc cacgggccgc cctttcctgg caggacagcg     1260

ggatcttgca gctgtcaggg gaggggaggc gggggctgat gtcaggaggg atacaaatag     1320

tgccgacggc tgggggccct gtctcccctc gccgcatcca ctctccggcc ggccgcctgc     1380

ccgccgcctc ctccgtgcgc ccgccagcct cgcccgcgcc gtcacc                    1426


<210>  23
<211>  798
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  MHCK7 promoter

<400>  23
tagttcatag cccatatatg gagttccgct agaagctgca tgtctaagct agacccttca       60

gattaaaaat aactgaggta agggcctggg taggggaggt ggtgtgagac gctcctgtct      120

ctcctctatc tgcccatcgg ccctttgggg aggaggaatg tgcccaagga ctaaaaaaag      180

gccatggagc cagaggggcg agggcaacag acctttcatg ggcaaacctt ggggccctgc      240

tgtctagcat gccccactac gggtctaggc tgcccatgta aggaggcaag gcctggggac      300

acccgagatg cctggttata attaacccag acatgtggct gccccccccc ccccaacacc      360

tgctgcctct aaaaataacc ctgtccctgg tggatcccct gcatgcgaag atcttcgaac      420

aaggctgtgg gggactgagg gcaggctgta acaggcttgg gggccagggc ttatacgtgc      480

ctgggactcc caaagtatta ctgttccatg ttcccggcga agggccagct gtcccccgcc      540

agctagactc agcacttagt ttaggaacca gtgagcaagt cagcccttgg ggcagcccat      600

acaaggccat ggggctgggc aagctgcacg cctgggtccg gggtgggcac ggtgcccggg      660

caacgagctg aaagctcatc tgctctcagg ggcccctccc tggggacagc ccctcctggc      720

tagtcacacc ctgtaggctc ctctatataa cccaggggca caggggctgc cctcattcta      780

ccaccacctc cacagcac                                                    798


