                         SEQUENCE LISTING

<110>  GENETHON
 
<120>  EXPRESSION CASSETTES FOR GENE THERAPY VECTORS

<130>  B2922PC00

<160>  12    

<170>  PatentIn version 3.3

<210>  1
<211>  507
<212>  DNA
<213>  artificial

<220>
<223>  human PGK promoter

<400>  1
ttggggttgc gccttttcca aggcagccct gggtttgcgc agggacgcgg ctgctctggg     60

cgtggttccg ggaaacgcag cggcgccgac cctgggtctc gcacattctt cacgtccgtt    120

cgcagcgtca cccggatctt cgccgctacc cttgtgggcc ccccggcgac gcttcctgct    180

ccgcccctaa gtcgggaagg ttccttgcgg ttcgcggcgt gccggacgtg acaaacggaa    240

gccgcacgtc tcactagtac cctcgcagac ggacagcgcc agggagcaat ggcagcgcgc    300

cgaccgcgat gggctgtggc caatagcggc tgctcagcag ggcgcgccga gagcagcggc    360

cgggaagggg cggtgcggga ggcggggtgt ggggcggtag tgtgggccct gttcctgccc    420

gcgcggtgtt ccgcattctg caagcctccg gagcgcacgt cggcagtcgg ctccctcgtt    480

gaccgaatca ccgacctctc tccccag                                        507


<210>  2
<211>  885
<212>  DNA
<213>  artificial

<220>
<223>  hSMN1 ORF

<400>  2
atggcgatga gcagcggcgg cagtggtggc ggcgtcccgg agcaggagga ttccgtgctg     60

ttccggcgcg gcacaggcca gagcgatgat tctgacattt gggatgatac agcactgata    120

aaagcatatg ataaagctgt ggcttcattt aagcatgctc taaagaatgg tgacatttgt    180

gaaacttcgg gtaaaccaaa aaccacacct aaaagaaaac ctgctaagaa gaataaaagc    240

caaaagaaga atactgcagc ttccttacaa cagtggaaag ttggggacaa atgttctgcc    300

atttggtcag aagacggttg catttaccca gctaccattg cttcaattga ttttaagaga    360

gaaacctgtg ttgtggttta cactggatat ggaaatagag aggagcaaaa tctgtccgat    420

ctactttccc caatctgtga agtagctaat aatatagaac aaaatgctca agagaatgaa    480

aatgaaagcc aagtttcaac agatgaaagt gagaactcca ggtctcctgg aaataaatca    540

gataacatca agcccaaatc tgctccatgg aactcttttc tccctccacc accccccatg    600

ccagggccaa gactgggacc aggaaagcca ggtctaaaat tcaatggccc accaccgcca    660

ccgccaccac caccacccca cttactatca tgctggctgc ctccatttcc ttctggacca    720

ccaataattc ccccaccacc tcccatatgt ccagattctc ttgatgatgc tgatgctttg    780

ggaagtatgt taatttcatg gtacatgagt ggctatcata ctggctatta tatgggtttc    840

agacaaaatc aaaaagaagg aaggtgctca cattccttaa attaa                    885


<210>  3
<211>  885
<212>  DNA
<213>  artificial

<220>
<223>  hSMN1co_ATUM

<400>  3
atggccatga gcagcggtgg ttcaggcggt ggagtgcctg agcaagagga ttcggtgctg     60

ttcaggaggg gcaccggaca gtccgacgac tccgatattt gggatgatac cgcactgatt    120

aaggcatacg acaaggccgt ggcgtccttc aagcacgcgc tgaagaatgg cgacatctgc    180

gaaacctcag gaaagcccaa gactaccccg aagcgcaaac cggccaagaa gaacaagtcg    240

cagaagaaga acactgccgc cagcctccaa cagtggaaag tcggggacaa gtgctccgcc    300

atctggtccg aggacggatg tatctacccg gccaccattg cctccatcga cttcaagcgc    360

gagacttgcg tggtcgtgta taccggatac ggcaaccgcg aagaacagaa tctcagcgat    420

ctgctgtcac ccatctgcga agtggcgaac aacatcgaac agaacgccca ggagaacgaa    480

aacgagtccc aagtctccac cgacgaatcc gagaactcga gatcacccgg gaacaagtcc    540

gacaacatta agccgaagtc tgccccctgg aactccttcc ttccgcctcc gccacctatg    600

cccggaccca gacttgggcc ggggaaacct ggtctgaagt tcaatggacc acctccgcct    660

cctccacctc ctcccccaca cctcctgtcc tgctggttgc ccccgtttcc ctccggaccg    720

cctattatcc caccaccgcc tcctatctgc ccggactccc tggacgatgc cgacgctctg    780

gggagcatgc tgatctcgtg gtacatgagc ggataccaca ccggctacta catgggattc    840

cggcagaacc agaaggaagg ccggtgttcg cattcgctga actga                    885


<210>  4
<211>  885
<212>  DNA
<213>  artificial

<220>
<223>  hSMN1co_Genc3

<400>  4
atggcaatga gcagcggagg aagcggagga ggagttcctg aacaggagga cagcgtgctg     60

ttcaggagag gaaccggaca gagcgacgac agtgacatct gggacgacac cgcactgatc    120

aaagcctacg acaaggcagt ggcaagcttt aagcacgccc tgaagaacgg agatatttgt    180

gagacaagcg gcaagcccaa aaccacaccc aaacgcaagc ccgctaagaa aaacaagtca    240

cagaagaaga acacagctgc ctcactgcag caatggaagg tgggagacaa gtgcagcgca    300

atctggagcg aggacggatg tatctacccc gcaacaatag ccagcatcga cttcaagaga    360

gaaacctgcg tggtggtgta caccggctac ggaaacagag aagagcagaa cctgagcgac    420

ctgctgagcc ctatatgcga ggtggctaat aacatcgagc aaaacgccca ggagaacgag    480

aacgagagcc aggttagcac cgatgagagc gaaaacagca gaagccccgg caacaaaagc    540

gacaacatca agcccaagag cgccccatgg aacagcttcc tgcctcctcc tccacctatg    600

cctggaccta gactgggacc aggaaaaccc ggactgaaat tcaacgggcc accccctcca    660

ccaccacctc ctcctcctca tctgctgtca tgctggctcc ctcctttccc ttccggacct    720

cctatcatcc cccctcctcc tcctatctgc cctgattctc tcgacgacgc cgacgctctg    780

ggatctatgc tgatctcctg gtacatgtcc ggctaccaca ccggttacta catgggcttc    840

agacagaacc aaaaggaagg ccggtgttcc cacagcctga actga                    885


<210>  5
<211>  885
<212>  DNA
<213>  artificial

<220>
<223>  hSMN1co_Genw4

<400>  5
atggccatgt cctccggagg aagcggagga ggcgtgcctg aacaggagga cagcgtgctg     60

tttaggaggg gcacaggcca gagcgacgac tccgacatct gggatgacac cgctctgatc    120

aaggcctacg acaaggccgt ggccagcttc aagcacgctc tgaagaacgg cgacatctgt    180

gagacctccg gcaagcccaa gaccacaccc aagaggaagc ccgccaagaa gaacaagtcc    240

cagaagaaga acaccgccgc ttccctgcag cagtggaagg tgggcgacaa gtgctccgct    300

atctggtccg aggatggctg catctacccc gccaccattg cctccatcga cttcaagagg    360

gagacctgcg tggtggtgta caccggctac ggcaacaggg aggagcagaa cctgagcgac    420

ctgctgagcc ctatctgcga ggtggctaac aacatcgagc agaacgccca agagaatgag    480

aacgagtccc aggtgagcac agacgagagc gagaattcca ggtcccccgg caataagagc    540

gacaacatca agcccaagag cgccccctgg aacagctttc tgcctcctcc cccccctatg    600

cctggcccta gactcggacc cggaaaaccc ggcctgaagt tcaacggacc tccccctcct    660

cctcctcctc ctcctcccca tctgctgagc tgctggctgc ccccttttcc ctccggacct    720

cccatcattc ctcctcctcc ccccatttgc cccgactccc tggacgatgc cgacgctctg    780

ggctccatgc tgatcagctg gtacatgtcc ggctaccaca ccggctacta catgggcttc    840

aggcagaacc agaaagaggg caggtgctcc cactccctga actga                    885


<210>  6
<211>  885
<212>  DNA
<213>  artificial

<220>
<223>  hSMN1co_MMV3.5

<400>  6
atggccatga gctctggagg gtctggagga ggagtgcctg agcaggagga ctctgtgctg     60

ttcagaagag gcacaggcca gtctgatgat tctgacatct gggatgacac agccctgatc    120

aaggcctatg acaaggctgt ggcttccttc aagcatgccc tgaagaacgg agacatctgt    180

gagacttctg gcaagccaaa gaccacaccc aagagaaagc ctgccaagaa gaacaagagc    240

cagaagaaga acactgctgc cagcctgcag cagtggaagg tgggggacaa gtgctctgct    300

atctggtcag aggatggctg tatctaccct gccaccattg ccagcattga cttcaagaga    360

gagacctgtg tggtggtgta cacaggctat ggcaacagag aggagcagaa cctgtctgac    420

ctgctgagcc ccatctgtga ggtggccaac aacattgagc agaatgccca ggagaatgag    480

aatgagagcc aggtgagcac agatgagtct gagaacagca gatctcctgg caacaagtct    540

gacaatatca agcccaagtc tgccccctgg aacagcttcc tgccccctcc tcctcctatg    600

cctggcccca gactgggacc tggcaagcct ggcctgaagt tcaacggccc ccctccccct    660

ccccctcccc ctccccctca cctgctgagc tgctggctgc cccccttccc ctctggcccc    720

cccatcatcc cccctcctcc ccctatctgc cctgactctc tggatgatgc tgatgccctg    780

ggcagcatgc tgatcagctg gtatatgtct ggctaccaca caggctacta catgggcttc    840

agacagaacc agaaggaggg cagatgcagc cacagcctga actga                    885


<210>  7
<211>  766
<212>  DNA
<213>  artificial

<220>
<223>  Human beta globin gene polyadenylation signal

<400>  7
attcacccca ccagtgcagg ctgcctatca gaaagtggtg gctggtgtgg ctaatgccct     60

ggcccacaag tatcactaag ctcgctttct tgctgtccaa tttctattaa aggttccttt    120

gttccctaag tccaactact aaactggggg atattatgaa gggccttgag catctggatt    180

ctgcctaata aaaaacattt attttcattg caatgatgta tttaaattat ttctgaatat    240

tttactaaaa agggaatgtg ggaggtcagt gcatttaaaa cataaagaaa tgaagagcta    300

gttcaaacct tgggaaaata cactatatct taaactccat gaaagaaggt gaggctgcaa    360

acagctaatg cacattggca acagccctga tgcctatgcc ttattcatcc ctcagaaaag    420

gattcaagta gaggcttgat ttggaggtta aagttttgct atgctgtatt ttacattact    480

tattgtttta gctgtcctca tgaatgtctt ttcactaccc atttgcttat cctgcatctc    540

tcagccttga ctccactcag ttctcttgct tagagatacc acctttcccc tgaagtgttc    600

cttccatgtt ttacggcgag atggtttctc ctcgcctggc cactcagcct tagttgtctc    660

tgttgtctta tagaggtcta cttgaagaag gaaaaacagg gggcatggtt tgactgtcct    720

gtgagccctt cttccctgcc tcccccactc acagtgaccc ggaatc                   766


<210>  8
<211>  574
<212>  DNA
<213>  artificial

<220>
<223>  Human beta globin gene polyadenylation signal - part

<400>  8
aaacatttat tttcattgca atgatgtatt taaattattt ctgaatattt tactaaaaag     60

ggaatgtggg aggtcagtgc atttaaaaca taaagaaatg aagagctagt tcaaaccttg    120

ggaaaataca ctatatctta aactccatga aagaaggtga ggctgcaaac agctaatgca    180

cattggcaac agccctgatg cctatgcctt attcatccct cagaaaagga ttcaagtaga    240

ggcttgattt ggaggttaaa gttttgctat gctgtatttt acattactta ttgttttagc    300

tgtcctcatg aatgtctttt cactacccat ttgcttatcc tgcatctctc agccttgact    360

ccactcagtt ctcttgctta gagataccac ctttcccctg aagtgttcct tccatgtttt    420

acggcgagat ggtttctcct cgcctggcca ctcagcctta gttgtctctg ttgtcttata    480

gaggtctact tgaagaagga aaaacagggg gcatggtttg actgtcctgt gagcccttct    540

tccctgcctc ccccactcac agtgacccgg aatc                                574


<210>  9
<211>  6
<212>  DNA
<213>  artificial

<220>
<223>  Kozak sequence

<400>  9
gccacc                                                                 6


<210>  10
<211>  49
<212>  DNA
<213>  artificial

<220>
<223>  synthetic polyA

<400>  10
aataaaagat ctttattttc attagatctg tgtgttggtt ttttgtgtg                 49


<210>  11
<211>  2610
<212>  DNA
<213>  artificial

<220>
<223>  7212 SMN expression cassette

<400>  11
ttggggttgc gccttttcca aggcagccct gggtttgcgc agggacgcgg ctgctctggg     60

cgtggttccg ggaaacgcag cggcgccgac cctgggtctc gcacattctt cacgtccgtt    120

cgcagcgtca cccggatctt cgccgctacc cttgtgggcc ccccggcgac gcttcctgct    180

ccgcccctaa gtcgggaagg ttccttgcgg ttcgcggcgt gccggacgtg acaaacggaa    240

gccgcacgtc tcactagtac cctcgcagac ggacagcgcc agggagcaat ggcagcgcgc    300

cgaccgcgat gggctgtggc caatagcggc tgctcagcag ggcgcgccga gagcagcggc    360

cgggaagggg cggtgcggga ggcggggtgt ggggcggtag tgtgggccct gttcctgccc    420

gcgcggtgtt ccgcattctg caagcctccg gagcgcacgt cggcagtcgg ctccctcgtt    480

gaccgaatca ccgacctctc tccccaggta cacatattga ccaaatcagg gtaattttgc    540

atttgtaatt ttaaaaaatg ctttcttctt ttaatatact tttttgttta tcttatttct    600

aatactttcc ctaatctctt tctttcaggg caataatgat acaatgtatc atgcctcttt    660

gcaccattct aaagaataac agtgataatt tctgggttaa ggcaatagca atatttctgc    720

atataaatat ttctgcatat aaattgtaac tgatgtaaga ggtttcatat tgctaatagc    780

agctacaatc cagctaccat tctgctttta ttttatggtt gggataaggc tggattattc    840

tgagtccaag ctaggccctt ttgctaatcc tgttcatacc tcttatcttc ctcccacagc    900

tcctgggcaa cgtgctggtc tgtgtgctgg cccatcactt tggcaaagaa ttcgccacca    960

tggcgatgag cagcggcggc agtggtggcg gcgtcccgga gcaggaggat tccgtgctgt   1020

tccggcgcgg cacaggccag agcgatgatt ctgacatttg ggatgataca gcactgataa   1080

aagcatatga taaagctgtg gcttcattta agcatgctct aaagaatggt gacatttgtg   1140

aaacttcggg taaaccaaaa accacaccta aaagaaaacc tgctaagaag aataaaagcc   1200

aaaagaagaa tactgcagct tccttacaac agtggaaagt tggggacaaa tgttctgcca   1260

tttggtcaga agacggttgc atttacccag ctaccattgc ttcaattgat tttaagagag   1320

aaacctgtgt tgtggtttac actggatatg gaaatagaga ggagcaaaat ctgtccgatc   1380

tactttcccc aatctgtgaa gtagctaata atatagaaca aaatgctcaa gagaatgaaa   1440

atgaaagcca agtttcaaca gatgaaagtg agaactccag gtctcctgga aataaatcag   1500

ataacatcaa gcccaaatct gctccatgga actcttttct ccctccacca ccccccatgc   1560

cagggccaag actgggacca ggaaagccag gtctaaaatt caatggccca ccaccgccac   1620

cgccaccacc accaccccac ttactatcat gctggctgcc tccatttcct tctggaccac   1680

caataattcc cccaccacct cccatatgtc cagattctct tgatgatgct gatgctttgg   1740

gaagtatgtt aatttcatgg tacatgagtg gctatcatac tggctattat atgggtttca   1800

gacaaaatca aaaagaagga aggtgctcac attccttaaa ttaaattcac cccaccagtg   1860

caggctgcct atcagaaagt ggtggctggt gtggctaatg ccctggccca caagtatcac   1920

taagctcgct ttcttgctgt ccaatttcta ttaaaggttc ctttgttccc taagtccaac   1980

tactaaactg ggggatatta tgaagggcct tgagcatctg gattctgcct aataaaaaac   2040

atttattttc attgcaatga tgtatttaaa ttatttctga atattttact aaaaagggaa   2100

tgtgggaggt cagtgcattt aaaacataaa gaaatgaaga gctagttcaa accttgggaa   2160

aatacactat atcttaaact ccatgaaaga aggtgaggct gcaaacagct aatgcacatt   2220

ggcaacagcc ctgatgccta tgccttattc atccctcaga aaaggattca agtagaggct   2280

tgatttggag gttaaagttt tgctatgctg tattttacat tacttattgt tttagctgtc   2340

ctcatgaatg tcttttcact acccatttgc ttatcctgca tctctcagcc ttgactccac   2400

tcagttctct tgcttagaga taccaccttt cccctgaagt gttccttcca tgttttacgg   2460

cgagatggtt tctcctcgcc tggccactca gccttagttg tctctgttgt cttatagagg   2520

tctacttgaa gaaggaaaaa cagggggcat ggtttgactg tcctgtgagc ccttcttccc   2580

tgcctccccc actcacagtg acccggaatc                                    2610


<210>  12
<211>  446
<212>  DNA
<213>  artificial

<220>
<223>  modified intron 2/exon 3 sequence from the human beta-globin gene

<400>  12
gtacacatat tgaccaaatc agggtaattt tgcatttgta attttaaaaa atgctttctt     60

cttttaatat acttttttgt ttatcttatt tctaatactt tccctaatct ctttctttca    120

gggcaataat gatacaatgt atcatgcctc tttgcaccat tctaaagaat aacagtgata    180

atttctgggt taaggcaata gcaatatttc tgcatataaa tatttctgca tataaattgt    240

aactgatgta agaggtttca tattgctaat agcagctaca atccagctac cattctgctt    300

ttattttatg gttgggataa ggctggatta ttctgagtcc aagctaggcc cttttgctaa    360

tcctgttcat acctcttatc ttcctcccac agctcctggg caacgtgctg gtctgtgtgc    420

tggcccatca ctttggcaaa gaattc                                         446


