                         SEQUENCE LISTING

<110>  Lysogene
 
<120>  COMPOSITIONS AND METHODS FOR TREATING GM1 GANGLIOSIDOSIS AND 
       OTHER DISORDERS

<130>  LYSO-004/01WO 

<150>  US 62/024,298
<151>  2020-05-13

<160>  6     

<170>  PatentIn version 3.5

<210>  1
<211>  2034
<212>  DNA
<213>  Homo sapiens

<400>  1
atgccggggt tcctggttcg catcctcctt ctgctgctgg ttctgctgct tctgggccct       60

acgcgcggct tgcgcaatgc cacccagagg atgtttgaaa ttgactatag ccgggactcc      120

ttcctcaagg atggccagcc atttcgctac atctcaggaa gcattcacta ctcccgtgtg      180

ccccgcttct actggaagga ccggctgctg aagatgaaga tggctgggct gaacgccatc      240

cagacgtatg tgccctggaa ctttcatgag ccctggccag gacagtacca gttttctgag      300

gaccatgatg tggaatattt tcttcggctg gctcatgagc tgggactgct ggttatcctg      360

aggcccgggc cctacatctg tgcagagtgg gaaatgggag gattacctgc ttggctgcta      420

gagaaagagt ctattcttct ccgctcctcc gacccagatt acctggcagc tgtggacaag      480

tggttgggag tccttctgcc caagatgaag cctctcctct atcagaatgg agggccagtt      540

ataacagtgc aggttgaaaa tgaatatggc agctactttg cctgtgattt tgactacctg      600

cgcttcctgc agaagcgctt tcgccaccat ctgggggatg atgtggttct gtttaccact      660

gatggagcac ataaaacatt cctgaaatgt ggggccctgc agggcctcta caccacggtg      720

gactttggaa caggcagcaa catcacagat gctttcctaa gccagaggaa gtgtgagccc      780

aaaggaccct tgatcaattc tgaattctat actggctggc tagatcactg gggccaacct      840

cactccacaa tcaagaccga agcagtggct tcctccctct atgatatact tgcccgtggg      900

gcgagtgtga acttgtacat gtttataggt gggaccaatt ttgcctattg gaatggggcc      960

aactcaccct atgcagcaca gcccaccagc tacgactatg atgccccact gagtgaggct     1020

ggggacctca ctgagaagta ttttgctctg cgaaacatca tccagaagtt tgaaaaagta     1080

ccagaaggtc ctatccctcc atctacacca aagtttgcat atggaaaggt cactttggaa     1140

aagttaaaga cagtgggagc agctctggac attctgtgtc cctctgggcc catcaaaagc     1200

ctttatccct tgacatttat ccaggtgaaa cagcattatg ggtttgtgct gtaccggaca     1260

acacttcctc aagattgcag caacccagca cctctctctt cacccctcaa tggagtccac     1320

gatcgagcat atgttgctgt ggatgggatc ccccagggag tccttgagcg aaacaatgtg     1380

atcactctga acataacagg gaaagctgga gccactctgg accttctggt agagaacatg     1440

ggacgtgtga actatggtgc atatatcaac gattttaagg gtttggtttc taacctgact     1500

ctcagttcca atatcctcac ggactggacg atctttccac tggacactga ggatgcagtg     1560

cgcagccacc tggggggctg gggacaccgt gacagtggcc accatgatga agcctgggcc     1620

cacaactcat ccaactacac gctcccggcc ttttatatgg ggaacttctc cattcccagt     1680

gggatcccag acttgcccca ggacaccttt atccagtttc ctggatggac caagggccag     1740

gtctggatta atggctttaa ccttggccgc tattggccag cccggggccc tcagttgacc     1800

ttgtttgtgc cccagcacat cctgatgacc tcggccccaa acaccatcac cgtgctggaa     1860

ctggagtggg caccctgcag cagtgatgat ccagaactat gtgctgtgac gttcgtggac     1920

aggccagtta ttggctcatc tgtgacctac gatcatccct ccaaacctgt tgaaaaaaga     1980

ctcatgcccc cacccccgca aaaaaacaaa gattcatggc tggaccatgt atga           2034


<210>  2
<211>  1751
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CAG Promoter

<400>  2
tgaattcggt acctagttat taatagtaat caattacggg gtcattagtt catagcccat       60

atatggagtt ccgcgttaca taacttacgg taaatggccc gcctggctga ccgcccaacg      120

acccccgccc attgacgtca ataatgacgt atgttcccat agtaacgcca atagggactt      180

tccattgacg tcaatgggtg gagtatttac ggtaaactgc ccacttggca gtacatcaag      240

tgtatcatat gccaagtacg ccccctattg acgtcaatga cggtaaatgg cccgcctggc      300

attatgccca gtacatgacc ttatgggact ttcctacttg gcagtacatc tacgtattag      360

tcatcgctat taccatggtc gaggtgagcc ccacgttctg cttcactctc cccatctccc      420

ccccctcccc acccccaatt ttgtatttat ttatttttta attattttgt gcagcgatgg      480

gggcgggggg gggggggggg cgcgcgccag gcggggcggg gcggggcgag gggcggggcg      540

gggcgaggcg gagaggtgcg gcggcagcca atcagagcgg cgcgctccga aagtttcctt      600

ttatggcgag gcggcggcgg cggcggccct ataaaaagcg aagcgcgcgg cgggcgggag      660

tcgctgcgcg ctgccttcgc cccgtgcccc gctccgccgc cgcctcgcgc cgcccgcccc      720

ggctctgact gaccgcgtta ctcccacagg tgagcgggcg ggacggccct tctcctccgg      780

gctgtaatta gcgcttggtt taatgacggc ttgtttcttt tctgtggctg cgtgaaagcc      840

ttgaggggct ccgggagggc cctttgtgcg gggggagcgg ctcggggggt gcgtgcgtgt      900

gtgtgtgcgt ggggagcgcc gcgtgcggct ccgcgctgcc cggcggctgt gagcgctgcg      960

ggcgcggcgc ggggctttgt gcgctccgca gtgtgcgcga ggggagcgcg gccgggggcg     1020

gtgccccgcg gtgcgggggg ggctgcgagg ggaacaaagg ctgcgtgcgg ggtgtgtgcg     1080

tgggggggtg agcagggggt gtgggcgcgt cggtcgggct gcaacccccc ctgcaccccc     1140

ctccccgagt tgctgagcac ggcccggctt cgggtgcggg gctccgtacg gggcgtggcg     1200

cggggctcgc cgtgccgggc ggggggtggc ggcaggtggg ggtgccgggc ggggcggggc     1260

cgcctcgggc cggggagggc tcgggggagg ggcgcggcgg cccccggagc gccggcggct     1320

gtcgaggcgc ggcgagccgc agccattgcc ttttatggta atcgtgcgag agggcgcagg     1380

gacttccttt gtcccaaatc tgtgcggagc cgaaatctgg gaggcgccgc cgcaccccct     1440

ctagcgggcg cggggcgaag cggtgcggcg ccggcaggaa ggaaatgggc ggggagggcc     1500

ttcgtgcgtc gccgcgccgc cgtccccttc tccctctcca gcctcggggc tgtccgcggg     1560

gggacggctg ccttcggggg ggacggggca gggcggggtt cggcttctgg cgtgtgaccg     1620

gcggctctag agcctctgct aaccatgttc atgccttctt ctttttccta cagctcctgg     1680

gcaacgtgct ggttattgtg ctgtctcatc attttggcaa agaattcgat atcaagcttg     1740

ctagcgccac c                                                          1751


<210>  3
<211>  510
<212>  DNA
<213>  Homo sapiens

<400>  3
acgggtggca tccctgtgac ccctccccag tgcctctcct ggccctggaa gttgccactc       60

cagtgcccac cagccttgtc ctaataaaat taagttgcat cattttgtct gactaggtgt      120

ccttctataa tattatgggg tggagggggg tggtatggag caaggggcaa gttgggaaga      180

caacctgtag ggcctgcggg gtctattggg aaccaagctg gagtgcagtg gcacaatctt      240

ggctcactgc aatctccgcc tcctgggttc aagcgattct cctgcctcag cctcccgagt      300

tgttgggatt ccaggcatgc atgaccaggc tcagctaatt tttgtttttt tggtagagac      360

ggggtttcac catattggcc aggctggtct ccaactccta atctcaggtg atctacccac      420

cttggcctcc caaattgctg ggattacagg cgtgaaccac tgctcccttc cctgtccttc      480

tgattttgta ggtaaccacg tgcggaccga                                       510


<210>  4
<211>  130
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Left Inverted Terminal Repeat Sequence

<400>  4
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct                                                             130


<210>  5
<211>  141
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Right Inverted Terminal Repeat Sequence

<400>  5
aggaacccct agtgatggag ttggccactc cctctctgcg cgctcgctcg ctcactgagg       60

ccgggcgacc aaaggtcgcc cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc      120

gagcgcgcag ctgcctgcag g                                                141


<210>  6
<211>  4600
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  LYS-GM101 Vector Construct

<400>  6
cctgcaggca gctgcgcgct cgctcgctca ctgaggccgc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggccaa ctccatcact      120

aggggttcct gcggccgcat cgattgaatt cggtacctag ttattaatag taatcaatta      180

cggggtcatt agttcatagc ccatatatgg agttccgcgt tacataactt acggtaaatg      240

gcccgcctgg ctgaccgccc aacgaccccc gcccattgac gtcaataatg acgtatgttc      300

ccatagtaac gccaataggg actttccatt gacgtcaatg ggtggagtat ttacggtaaa      360

ctgcccactt ggcagtacat caagtgtatc atatgccaag tacgccccct attgacgtca      420

atgacggtaa atggcccgcc tggcattatg cccagtacat gaccttatgg gactttccta      480

cttggcagta catctacgta ttagtcatcg ctattaccat ggtcgaggtg agccccacgt      540

tctgcttcac tctccccatc tcccccccct ccccaccccc aattttgtat ttatttattt      600

tttaattatt ttgtgcagcg atgggggcgg gggggggggg ggggcgcgcg ccaggcgggg      660

cggggcgggg cgaggggcgg ggcggggcga ggcggagagg tgcggcggca gccaatcaga      720

gcggcgcgct ccgaaagttt ccttttatgg cgaggcggcg gcggcggcgg ccctataaaa      780

agcgaagcgc gcggcgggcg ggagtcgctg cgcgctgcct tcgccccgtg ccccgctccg      840

ccgccgcctc gcgccgcccg ccccggctct gactgaccgc gttactccca caggtgagcg      900

ggcgggacgg cccttctcct ccgggctgta attagcgctt ggtttaatga cggcttgttt      960

cttttctgtg gctgcgtgaa agccttgagg ggctccggga gggccctttg tgcgggggga     1020

gcggctcggg gggtgcgtgc gtgtgtgtgt gcgtggggag cgccgcgtgc ggctccgcgc     1080

tgcccggcgg ctgtgagcgc tgcgggcgcg gcgcggggct ttgtgcgctc cgcagtgtgc     1140

gcgaggggag cgcggccggg ggcggtgccc cgcggtgcgg ggggggctgc gaggggaaca     1200

aaggctgcgt gcggggtgtg tgcgtggggg ggtgagcagg gggtgtgggc gcgtcggtcg     1260

ggctgcaacc ccccctgcac ccccctcccc gagttgctga gcacggcccg gcttcgggtg     1320

cggggctccg tacggggcgt ggcgcggggc tcgccgtgcc gggcgggggg tggcggcagg     1380

tgggggtgcc gggcggggcg gggccgcctc gggccgggga gggctcgggg gaggggcgcg     1440

gcggcccccg gagcgccggc ggctgtcgag gcgcggcgag ccgcagccat tgccttttat     1500

ggtaatcgtg cgagagggcg cagggacttc ctttgtccca aatctgtgcg gagccgaaat     1560

ctgggaggcg ccgccgcacc ccctctagcg ggcgcggggc gaagcggtgc ggcgccggca     1620

ggaaggaaat gggcggggag ggccttcgtg cgtcgccgcg ccgccgtccc cttctccctc     1680

tccagcctcg gggctgtccg cggggggacg gctgccttcg ggggggacgg ggcagggcgg     1740

ggttcggctt ctggcgtgtg accggcggct ctagagcctc tgctaaccat gttcatgcct     1800

tcttcttttt cctacagctc ctgggcaacg tgctggttat tgtgctgtct catcattttg     1860

gcaaagaatt cgatatcaag cttgctagcg ccaccatgcc ggggttcctg gttcgcatcc     1920

tccttctgct gctggttctg ctgcttctgg gccctacgcg cggcttgcgc aatgccaccc     1980

agaggatgtt tgaaattgac tatagccggg actccttcct caaggatggc cagccatttc     2040

gctacatctc aggaagcatt cactactccc gtgtgccccg cttctactgg aaggaccggc     2100

tgctgaagat gaagatggct gggctgaacg ccatccagac gtatgtgccc tggaactttc     2160

atgagccctg gccaggacag taccagtttt ctgaggacca tgatgtggaa tattttcttc     2220

ggctggctca tgagctggga ctgctggtta tcctgaggcc cgggccctac atctgtgcag     2280

agtgggaaat gggaggatta cctgcttggc tgctagagaa agagtctatt cttctccgct     2340

cctccgaccc agattacctg gcagctgtgg acaagtggtt gggagtcctt ctgcccaaga     2400

tgaagcctct cctctatcag aatggagggc cagttataac agtgcaggtt gaaaatgaat     2460

atggcagcta ctttgcctgt gattttgact acctgcgctt cctgcagaag cgctttcgcc     2520

accatctggg ggatgatgtg gttctgttta ccactgatgg agcacataaa acattcctga     2580

aatgtggggc cctgcagggc ctctacacca cggtggactt tggaacaggc agcaacatca     2640

cagatgcttt cctaagccag aggaagtgtg agcccaaagg acccttgatc aattctgaat     2700

tctatactgg ctggctagat cactggggcc aacctcactc cacaatcaag accgaagcag     2760

tggcttcctc cctctatgat atacttgccc gtggggcgag tgtgaacttg tacatgttta     2820

taggtgggac caattttgcc tattggaatg gggccaactc accctatgca gcacagccca     2880

ccagctacga ctatgatgcc ccactgagtg aggctgggga cctcactgag aagtattttg     2940

ctctgcgaaa catcatccag aagtttgaaa aagtaccaga aggtcctatc cctccatcta     3000

caccaaagtt tgcatatgga aaggtcactt tggaaaagtt aaagacagtg ggagcagctc     3060

tggacattct gtgtccctct gggcccatca aaagccttta tcccttgaca tttatccagg     3120

tgaaacagca ttatgggttt gtgctgtacc ggacaacact tcctcaagat tgcagcaacc     3180

cagcacctct ctcttcaccc ctcaatggag tccacgatcg agcatatgtt gctgtggatg     3240

ggatccccca gggagtcctt gagcgaaaca atgtgatcac tctgaacata acagggaaag     3300

ctggagccac tctggacctt ctggtagaga acatgggacg tgtgaactat ggtgcatata     3360

tcaacgattt taagggtttg gtttctaacc tgactctcag ttccaatatc ctcacggact     3420

ggacgatctt tccactggac actgaggatg cagtgcgcag ccacctgggg ggctggggac     3480

accgtgacag tggccaccat gatgaagcct gggcccacaa ctcatccaac tacacgctcc     3540

cggcctttta tatggggaac ttctccattc ccagtgggat cccagacttg ccccaggaca     3600

cctttatcca gtttcctgga tggaccaagg gccaggtctg gattaatggc tttaaccttg     3660

gccgctattg gccagcccgg ggccctcagt tgaccttgtt tgtgccccag cacatcctga     3720

tgacctcggc cccaaacacc atcaccgtgc tggaactgga gtgggcaccc tgcagcagtg     3780

atgatccaga actatgtgct gtgacgttcg tggacaggcc agttattggc tcatctgtga     3840

cctacgatca tccctccaaa cctgttgaaa aaagactcat gcccccaccc ccgcaaaaaa     3900

acaaagattc atggctggac catgtatgac tcgagagatc tacgggtggc atccctgtga     3960

cccctcccca gtgcctctcc tggccctgga agttgccact ccagtgccca ccagccttgt     4020

cctaataaaa ttaagttgca tcattttgtc tgactaggtg tccttctata atattatggg     4080

gtggaggggg gtggtatgga gcaaggggca agttgggaag acaacctgta gggcctgcgg     4140

ggtctattgg gaaccaagct ggagtgcagt ggcacaatct tggctcactg caatctccgc     4200

ctcctgggtt caagcgattc tcctgcctca gcctcccgag ttgttgggat tccaggcatg     4260

catgaccagg ctcagctaat ttttgttttt ttggtagaga cggggtttca ccatattggc     4320

caggctggtc tccaactcct aatctcaggt gatctaccca ccttggcctc ccaaattgct     4380

gggattacag gcgtgaacca ctgctccctt ccctgtcctt ctgattttgt aggtaaccac     4440

gtgcggaccg agcggccgca ggaaccccta gtgatggagt tggccactcc ctctctgcgc     4500

gctcgctcgc tcactgaggc cgggcgacca aaggtcgccc gacgcccggg ctttgcccgg     4560

gcggcctcag tgagcgagcg agcgcgcagc tgcctgcagg                           4600


