                         SEQUENCE LISTING

<110>  4D Molecular Therapeutics Inc.
 
<120>  Codon Optimized REP1 Genes and Uses Thereof

<130>  090400-5011 WO

<150>  US 63/073,837
<151>  2020-09-02

<160>  9     

<170>  PatentIn version 3.5

<210>  1
<211>  1962
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  codon optimized human REP1

<400>  1
atggctgata cactgccttc tgagtttgat gtgatcgtga ttggaactgg actgcctgag       60

agtattattg ctgctgcttg tagtagaagc ggccggagag tgctgcacgt ggacagcaga      120

tcctactatg gcggcaactg ggcctctttc agcttttccg gcctgctgag ctggctgaag      180

gagtaccagg agaactccga catcgtgtct gatagccccg tgtggcagga ccagatcctg      240

gagaatgagg aggccatcgc cctgtccagg aaggataaga ccatccagca cgtggaggtg      300

ttctgctatg ccagccagga cctgcacgag gatgtggagg aggcaggcgc cctgcagaag      360

aaccacgccc tggtgacctc cgccaattct acagaggccg ccgactccgc ctttctgcct      420

accgaggatg agtccctgtc tacaatgtct tgtgagatgc tgaccgagca gacacctagc      480

tccgatccag agaacgccct ggaggtcaat ggcgccgagg tgaccggcga gaaggagaac      540

cactgcgacg ataagacctg cgtgccaagc acatccgccg aggacatgtc cgagaacgtg      600

cctatcgccg aggataccac agagcagcca aagaagaatc gcatcacata cagccagatc      660

atcaaggagg gcaggcgctt caatatcgac ctggtgtcta agctgctgta cagccggggc      720

ctgctgatcg atctgctgat caagagcaac gtgtcccgct atgccgagtt caagaatatc      780

accagaatcc tggcctttcg ggagggaaga gtggagcagg tgccctgcag cagagccgac      840

gtgttcaact ccaagcagct gacaatggtg gagaagagga tgctgatgaa gttcctgaca      900

ttttgtatgg agtacgagaa gtatccagat gagtacaagg gctatgagga gatcaccttt      960

tacgagtatc tgaagaccca gaagctgaca cccaatctgc agtacatcgt gatgcactcc     1020

atcgccatga cctctgagac agcctctagc accatcgacg gcctgaaggc cacaaagaac     1080

ttcctgcact gcctgggccg gtacggcaat acacccttcc tgtttcctct gtatggccag     1140

ggcgagctgc cccagtgctt ctgtagaatg tgcgccgtgt ttggcggcat ctattgcctg     1200

aggcactctg tgcagtgtct ggtggtggac aaggagagcc gcaagtgtaa ggccatcatc     1260

gatcagtttg gccagcggat catctctgag cacttcctgg tggaggacag ctactttcct     1320

gagaacatgt gctccagggt gcagtatcgc cagatcagcc gggccgtgct gatcaccgat     1380

agatccgtgc tgaagacaga cagcgatcag cagatcagca tcctgaccgt gccagcagag     1440

gagccaggca ccttcgccgt gagagtgatc gagctgtgct cctctaccat gacatgtatg     1500

aagggcacct acctggtgca cctgacctgc acaagctcca agacagcccg cgaggacctg     1560

gagagcgtgg tgcagaagct gttcgtgccc tacaccgaga tggagatcga gaacgagcag     1620

gtggagaagc ctagaatcct gtgggccctg tacttcaaca tgagagactc tagcgatatc     1680

tctaggagct gttacaacga tctgccctct aacgtgtacg tgtgcagcgg acctgactgt     1740

ggcctgggaa acgataatgc cgtgaagcag gccgagacac tgttccagga gatttgccct     1800

aacgaggact tttgtccccc tccacccaat ccagaggata tcatcctgga cggcgattcc     1860

ctgcagccag aggcctctga gtcctctgcc atccccgagg ccaatagcga aacattcaaa     1920

gaaagcacaa atctgggaaa cctggaagaa agtagtgagt aa                        1962


<210>  2
<211>  653
<212>  PRT
<213>  Homo sapiens

<400>  2

Met Ala Asp Thr Leu Pro Ser Glu Phe Asp Val Ile Val Ile Gly Thr 
1               5                   10                  15      


Gly Leu Pro Glu Ser Ile Ile Ala Ala Ala Cys Ser Arg Ser Gly Arg 
            20                  25                  30          


Arg Val Leu His Val Asp Ser Arg Ser Tyr Tyr Gly Gly Asn Trp Ala 
        35                  40                  45              


Ser Phe Ser Phe Ser Gly Leu Leu Ser Trp Leu Lys Glu Tyr Gln Glu 
    50                  55                  60                  


Asn Ser Asp Ile Val Ser Asp Ser Pro Val Trp Gln Asp Gln Ile Leu 
65                  70                  75                  80  


Glu Asn Glu Glu Ala Ile Ala Leu Ser Arg Lys Asp Lys Thr Ile Gln 
                85                  90                  95      


His Val Glu Val Phe Cys Tyr Ala Ser Gln Asp Leu His Glu Asp Val 
            100                 105                 110         


Glu Glu Ala Gly Ala Leu Gln Lys Asn His Ala Leu Val Thr Ser Ala 
        115                 120                 125             


Asn Ser Thr Glu Ala Ala Asp Ser Ala Phe Leu Pro Thr Glu Asp Glu 
    130                 135                 140                 


Ser Leu Ser Thr Met Ser Cys Glu Met Leu Thr Glu Gln Thr Pro Ser 
145                 150                 155                 160 


Ser Asp Pro Glu Asn Ala Leu Glu Val Asn Gly Ala Glu Val Thr Gly 
                165                 170                 175     


Glu Lys Glu Asn His Cys Asp Asp Lys Thr Cys Val Pro Ser Thr Ser 
            180                 185                 190         


Ala Glu Asp Met Ser Glu Asn Val Pro Ile Ala Glu Asp Thr Thr Glu 
        195                 200                 205             


Gln Pro Lys Lys Asn Arg Ile Thr Tyr Ser Gln Ile Ile Lys Glu Gly 
    210                 215                 220                 


Arg Arg Phe Asn Ile Asp Leu Val Ser Lys Leu Leu Tyr Ser Arg Gly 
225                 230                 235                 240 


Leu Leu Ile Asp Leu Leu Ile Lys Ser Asn Val Ser Arg Tyr Ala Glu 
                245                 250                 255     


Phe Lys Asn Ile Thr Arg Ile Leu Ala Phe Arg Glu Gly Arg Val Glu 
            260                 265                 270         


Gln Val Pro Cys Ser Arg Ala Asp Val Phe Asn Ser Lys Gln Leu Thr 
        275                 280                 285             


Met Val Glu Lys Arg Met Leu Met Lys Phe Leu Thr Phe Cys Met Glu 
    290                 295                 300                 


Tyr Glu Lys Tyr Pro Asp Glu Tyr Lys Gly Tyr Glu Glu Ile Thr Phe 
305                 310                 315                 320 


Tyr Glu Tyr Leu Lys Thr Gln Lys Leu Thr Pro Asn Leu Gln Tyr Ile 
                325                 330                 335     


Val Met His Ser Ile Ala Met Thr Ser Glu Thr Ala Ser Ser Thr Ile 
            340                 345                 350         


Asp Gly Leu Lys Ala Thr Lys Asn Phe Leu His Cys Leu Gly Arg Tyr 
        355                 360                 365             


Gly Asn Thr Pro Phe Leu Phe Pro Leu Tyr Gly Gln Gly Glu Leu Pro 
    370                 375                 380                 


Gln Cys Phe Cys Arg Met Cys Ala Val Phe Gly Gly Ile Tyr Cys Leu 
385                 390                 395                 400 


Arg His Ser Val Gln Cys Leu Val Val Asp Lys Glu Ser Arg Lys Cys 
                405                 410                 415     


Lys Ala Ile Ile Asp Gln Phe Gly Gln Arg Ile Ile Ser Glu His Phe 
            420                 425                 430         


Leu Val Glu Asp Ser Tyr Phe Pro Glu Asn Met Cys Ser Arg Val Gln 
        435                 440                 445             


Tyr Arg Gln Ile Ser Arg Ala Val Leu Ile Thr Asp Arg Ser Val Leu 
    450                 455                 460                 


Lys Thr Asp Ser Asp Gln Gln Ile Ser Ile Leu Thr Val Pro Ala Glu 
465                 470                 475                 480 


Glu Pro Gly Thr Phe Ala Val Arg Val Ile Glu Leu Cys Ser Ser Thr 
                485                 490                 495     


Met Thr Cys Met Lys Gly Thr Tyr Leu Val His Leu Thr Cys Thr Ser 
            500                 505                 510         


Ser Lys Thr Ala Arg Glu Asp Leu Glu Ser Val Val Gln Lys Leu Phe 
        515                 520                 525             


Val Pro Tyr Thr Glu Met Glu Ile Glu Asn Glu Gln Val Glu Lys Pro 
    530                 535                 540                 


Arg Ile Leu Trp Ala Leu Tyr Phe Asn Met Arg Asp Ser Ser Asp Ile 
545                 550                 555                 560 


Ser Arg Ser Cys Tyr Asn Asp Leu Pro Ser Asn Val Tyr Val Cys Ser 
                565                 570                 575     


Gly Pro Asp Cys Gly Leu Gly Asn Asp Asn Ala Val Lys Gln Ala Glu 
            580                 585                 590         


Thr Leu Phe Gln Glu Ile Cys Pro Asn Glu Asp Phe Cys Pro Pro Pro 
        595                 600                 605             


Pro Asn Pro Glu Asp Ile Ile Leu Asp Gly Asp Ser Leu Gln Pro Glu 
    610                 615                 620                 


Ala Ser Glu Ser Ser Ala Ile Pro Glu Ala Asn Ser Glu Thr Phe Lys 
625                 630                 635                 640 


Glu Ser Thr Asn Leu Gly Asn Leu Glu Glu Ser Ser Glu 
                645                 650             


<210>  3
<211>  1962
<212>  DNA
<213>  Homo sapiens

<400>  3
atggcggata ctctcccttc ggagtttgat gtgatcgtaa tagggacggg tttgcctgaa       60

tccatcattg cagctgcatg ttcaagaagt ggccggagag ttctgcatgt tgattcaaga      120

agctactatg gaggaaactg ggccagtttt agcttttcag gactattgtc ctggctaaag      180

gaataccagg aaaacagtga cattgtaagt gacagtccag tgtggcaaga ccagatcctt      240

gaaaatgaag aagccattgc tcttagcagg aaggacaaaa ctattcaaca tgtggaagta      300

ttttgttatg ccagtcagga tttgcatgaa gatgtcgaag aagctggtgc actgcagaaa      360

aatcatgctc ttgtgacatc tgcaaactcc acagaagctg cagattctgc cttcctgcct      420

acggaggatg agtcattaag cactatgagc tgtgaaatgc tcacagaaca aactccaagc      480

agcgatccag agaatgcgct agaagtaaat ggtgctgaag tgacagggga aaaagaaaac      540

cattgtgatg ataaaacttg tgtgccatca acttcagcag aagacatgag tgaaaatgtg      600

cctatagcag aagataccac agagcaacca aagaaaaaca gaattactta ctcacaaatt      660

attaaagaag gcaggagatt taatattgat ttagtatcaa agctgctgta ttctcgagga      720

ttactaattg atcttctaat caaatctaat gttagtcgat atgcagagtt taaaaatatt      780

accaggattc ttgcatttcg agaaggacga gtggaacagg ttccgtgttc cagagcagat      840

gtctttaata gcaaacaact tactatggta gaaaagcgaa tgctaatgaa atttcttaca      900

ttttgtatgg aatatgagaa atatcctgat gaatataaag gatatgaaga gatcacattt      960

tatgaatatt taaagactca aaaattaacc cccaacctcc aatatattgt catgcattca     1020

attgcaatga catcagagac agccagcagc accatagatg gtctcaaagc taccaaaaac     1080

tttcttcact gtcttgggcg gtatggcaac actccatttt tgtttccttt atatggccaa     1140

ggagaactcc cccagtgttt ctgcaggatg tgtgctgtgt ttggtggaat ttattgtctt     1200

cgccattcag tacagtgcct tgtagtggac aaagaatcca gaaaatgtaa agcaattata     1260

gatcagtttg gtcagagaat aatctctgag catttcctcg tggaggacag ttactttcct     1320

gagaacatgt gctcacgtgt gcaatacagg cagatctcca gggcagtgct gattacagat     1380

agatctgtcc taaaaacaga ttcagatcaa cagatttcca ttttgacagt gccagcagag     1440

gaaccaggaa cttttgctgt tcgggtcatt gagttatgtt cttcaacgat gacatgcatg     1500

aaaggcacct atttggttca tttgacttgc acatcttcta aaacagcaag agaagattta     1560

gaatcagttg tgcagaaatt gtttgttcca tatactgaaa tggagataga aaatgaacaa     1620

gtagaaaagc caagaattct gtgggctctt tacttcaata tgagagattc gtcagacatc     1680

agcaggagct gttataatga tttaccatcc aacgtttatg tctgctctgg cccagattgt     1740

ggtttaggaa atgataatgc agtcaaacag gctgaaacac ttttccagga aatctgcccc     1800

aatgaagatt tctgtccccc tccaccaaat cctgaagaca ttatccttga tggagacagt     1860

ttacagccag aggcttcaga atccagtgcc ataccagagg ctaactcgga gactttcaag     1920

gaaagcacaa accttggaaa cctagaggag tcctctgaat aa                        1962


<210>  4
<211>  1664
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  CAG promoter

<400>  4
actagttatt aatagtaatc aattacgggg tcattagttc atagcccata tatggagttc       60

cgcgttacat aacttacggt aaatggcccg cctggctgac cgcccaacga cccccgccca      120

ttgacgtcaa taatgacgta tgttcccata gtaacgccaa tagggacttt ccattgacgt      180

caatgggtgg agtatttacg gtaaactgcc cacttggcag tacatcaagt gtatcatatg      240

ccaagtacgc cccctattga cgtcaatgac ggtaaatggc ccgcctggca ttatgcccag      300

tacatgacct tatgggactt tcctacttgg cagtacatct acgtattagt catcgctatt      360

accatggtcg aggtgagccc cacgttctgc ttcactctcc ccatctcccc cccctcccca      420

cccccaattt tgtatttatt tattttttaa ttattttgtg cagcgatggg ggcggggggg      480

gggggggggc gcgcgccagg cggggcgggg cggggcgagg ggcggggcgg ggcgaggcgg      540

agaggtgcgg cggcagccaa tcagagcggc gcgctccgaa agtttccttt tatggcgagg      600

cggcggcggc ggcggcccta taaaaagcga agcgcgcggc gggcggggag tcgctgcgac      660

gctgccttcg ccccgtgccc cgctccgccg ccgcctcgcg ccgcccgccc cggctctgac      720

tgaccgcgtt actcccacag gtgagcgggc gggacggccc ttctcctccg ggctgtaatt      780

agcgcttggt ttaatgacgg cttgtttctt ttctgtggct gcgtgaaagc cttgaggggc      840

tccgggaggg ccctttgtgc ggggggagcg gctcgggggg tgcgtgcgtg tgtgtgtgcg      900

tggggagcgc cgcgtgcggc tccgcgctgc ccggcggctg tgagcgctgc gggcgcggcg      960

cggggctttg tgcgctccgc agtgtgcgcg aggggagcgc ggccgggggc ggtgccccgc     1020

ggtgcggggg gggctgcgag gggaacaaag gctgcgtgcg gggtgtgtgc gtgggggggt     1080

gagcaggggg tgtgggcgcg tcggtcgggc tgcaaccccc cctgcacccc cctccccgag     1140

ttgctgagca cggcccggct tcgggtgcgg ggctccgtac ggggcgtggc gcggggctcg     1200

ccgtgccggg cggggggtgg cggcaggtgg gggtgccggg cggggcgggg ccgcctcggg     1260

ccggggaggg ctcgggggag gggcgcggcg gcccccggag cgccggcggc tgtcgaggcg     1320

cggcgagccg cagccattgc cttttatggt aatcgtgcga gagggcgcag ggacttcctt     1380

tgtcccaaat ctgtgcggag ccgaaatctg ggaggcgccg ccgcaccccc tctagcgggc     1440

gcggggcgaa gcggtgcggc gccggcagga aggaaatggg cggggagggc cttcgtgcgt     1500

cgccgcgccg ccgtcccctt ctccctctcc agcctcgggg ctgtccgcgg ggggacggct     1560

gccttcgggg gggacggggc agggcggggt tcggcttctg gcgtgtgacc ggcggctcta     1620

gagcctctgc taaccatgtt catgccttct tctttttcct acag                      1664


<210>  5
<211>  4271
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  REP1 transgene cassette

<400>  5
ttggccactc cctctctgcg cgctcgctcg ctcactgagg ccgggcgacc aaaggtcgcc       60

cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc gagcgcgcag agagggagtg      120

gccaactcca tcactagggg ttcctatcga ttgaattccc cggggatcca ctagttatta      180

atagtaatca attacggggt cattagttca tagcccatat atggagttcc gcgttacata      240

acttacggta aatggcccgc ctggctgacc gcccaacgac ccccgcccat tgacgtcaat      300

aatgacgtat gttcccatag taacgccaat agggactttc cattgacgtc aatgggtgga      360

gtatttacgg taaactgccc acttggcagt acatcaagtg tatcatatgc caagtacgcc      420

ccctattgac gtcaatgacg gtaaatggcc cgcctggcat tatgcccagt acatgacctt      480

atgggacttt cctacttggc agtacatcta cgtattagtc atcgctatta ccatggtcga      540

ggtgagcccc acgttctgct tcactctccc catctccccc ccctccccac ccccaatttt      600

gtatttattt attttttaat tattttgtgc agcgatgggg gcgggggggg ggggggggcg      660

cgcgccaggc ggggcggggc ggggcgaggg gcggggcggg gcgaggcgga gaggtgcggc      720

ggcagccaat cagagcggcg cgctccgaaa gtttcctttt atggcgaggc ggcggcggcg      780

gcggccctat aaaaagcgaa gcgcgcggcg ggcggggagt cgctgcgacg ctgccttcgc      840

cccgtgcccc gctccgccgc cgcctcgcgc cgcccgcccc ggctctgact gaccgcgtta      900

ctcccacagg tgagcgggcg ggacggccct tctcctccgg gctgtaatta gcgcttggtt      960

taatgacggc ttgtttcttt tctgtggctg cgtgaaagcc ttgaggggct ccgggagggc     1020

cctttgtgcg gggggagcgg ctcggggggt gcgtgcgtgt gtgtgtgcgt ggggagcgcc     1080

gcgtgcggct ccgcgctgcc cggcggctgt gagcgctgcg ggcgcggcgc ggggctttgt     1140

gcgctccgca gtgtgcgcga ggggagcgcg gccgggggcg gtgccccgcg gtgcgggggg     1200

ggctgcgagg ggaacaaagg ctgcgtgcgg ggtgtgtgcg tgggggggtg agcagggggt     1260

gtgggcgcgt cggtcgggct gcaacccccc ctgcaccccc ctccccgagt tgctgagcac     1320

ggcccggctt cgggtgcggg gctccgtacg gggcgtggcg cggggctcgc cgtgccgggc     1380

ggggggtggc ggcaggtggg ggtgccgggc ggggcggggc cgcctcgggc cggggagggc     1440

tcgggggagg ggcgcggcgg cccccggagc gccggcggct gtcgaggcgc ggcgagccgc     1500

agccattgcc ttttatggta atcgtgcgag agggcgcagg gacttccttt gtcccaaatc     1560

tgtgcggagc cgaaatctgg gaggcgccgc cgcaccccct ctagcgggcg cggggcgaag     1620

cggtgcggcg ccggcaggaa ggaaatgggc ggggagggcc ttcgtgcgtc gccgcgccgc     1680

cgtccccttc tccctctcca gcctcggggc tgtccgcggg gggacggctg ccttcggggg     1740

ggacggggca gggcggggtt cggcttctgg cgtgtgaccg gcggctctag agcctctgct     1800

aaccatgttc atgccttctt ctttttccta cagtctagag tcgacctgca gaagcttcca     1860

ccatggctga tacactgcct tctgagtttg atgtgatcgt gattggaact ggactgcctg     1920

agagtattat tgctgctgct tgtagtagaa gcggccggag agtgctgcac gtggacagca     1980

gatcctacta tggcggcaac tgggcctctt tcagcttttc cggcctgctg agctggctga     2040

aggagtacca ggagaactcc gacatcgtgt ctgatagccc cgtgtggcag gaccagatcc     2100

tggagaatga ggaggccatc gccctgtcca ggaaggataa gaccatccag cacgtggagg     2160

tgttctgcta tgccagccag gacctgcacg aggatgtgga ggaggcaggc gccctgcaga     2220

agaaccacgc cctggtgacc tccgccaatt ctacagaggc cgccgactcc gcctttctgc     2280

ctaccgagga tgagtccctg tctacaatgt cttgtgagat gctgaccgag cagacaccta     2340

gctccgatcc agagaacgcc ctggaggtca atggcgccga ggtgaccggc gagaaggaga     2400

accactgcga cgataagacc tgcgtgccaa gcacatccgc cgaggacatg tccgagaacg     2460

tgcctatcgc cgaggatacc acagagcagc caaagaagaa tcgcatcaca tacagccaga     2520

tcatcaagga gggcaggcgc ttcaatatcg acctggtgtc taagctgctg tacagccggg     2580

gcctgctgat cgatctgctg atcaagagca acgtgtcccg ctatgccgag ttcaagaata     2640

tcaccagaat cctggccttt cgggagggaa gagtggagca ggtgccctgc agcagagccg     2700

acgtgttcaa ctccaagcag ctgacaatgg tggagaagag gatgctgatg aagttcctga     2760

cattttgtat ggagtacgag aagtatccag atgagtacaa gggctatgag gagatcacct     2820

tttacgagta tctgaagacc cagaagctga cacccaatct gcagtacatc gtgatgcact     2880

ccatcgccat gacctctgag acagcctcta gcaccatcga cggcctgaag gccacaaaga     2940

acttcctgca ctgcctgggc cggtacggca atacaccctt cctgtttcct ctgtatggcc     3000

agggcgagct gccccagtgc ttctgtagaa tgtgcgccgt gtttggcggc atctattgcc     3060

tgaggcactc tgtgcagtgt ctggtggtgg acaaggagag ccgcaagtgt aaggccatca     3120

tcgatcagtt tggccagcgg atcatctctg agcacttcct ggtggaggac agctactttc     3180

ctgagaacat gtgctccagg gtgcagtatc gccagatcag ccgggccgtg ctgatcaccg     3240

atagatccgt gctgaagaca gacagcgatc agcagatcag catcctgacc gtgccagcag     3300

aggagccagg caccttcgcc gtgagagtga tcgagctgtg ctcctctacc atgacatgta     3360

tgaagggcac ctacctggtg cacctgacct gcacaagctc caagacagcc cgcgaggacc     3420

tggagagcgt ggtgcagaag ctgttcgtgc cctacaccga gatggagatc gagaacgagc     3480

aggtggagaa gcctagaatc ctgtgggccc tgtacttcaa catgagagac tctagcgata     3540

tctctaggag ctgttacaac gatctgccct ctaacgtgta cgtgtgcagc ggacctgact     3600

gtggcctggg aaacgataat gccgtgaagc aggccgagac actgttccag gagatttgcc     3660

ctaacgagga cttttgtccc cctccaccca atccagagga tatcatcctg gacggcgatt     3720

ccctgcagcc agaggcctct gagtcctctg ccatccccga ggccaatagc gaaacattca     3780

aagaaagcac aaatctggga aacctggaag aaagtagtga gtaagcctcg agcagcgctg     3840

ctcgagagat ctgcggccgc gagctcgggg atccagacat gataagatac attgatgagt     3900

ttggacaaac cacaactaga atgcagtgaa aaaaatgctt tatttgtgaa atttgtgatg     3960

ctattgcttt atttgtaacc attataagct gcaataaaca agttaacaac aacaattgca     4020

ttcattttat gtttcaggtt cagggggagg tgtgggaggt tttttaaagc aagtaaaacc     4080

tctacaaatg tggtatggct gattatgatc aatgcatcct agccggagga acccctagtg     4140

atggagttgg ccactccctc tctgcgcgct cgctcgctca ctgaggccgc ccgggcaaag     4200

cccgggcgtc gggcgacctt tggtcgcccg gcctcagtga gcgagcgagc gcgcagagag     4260

ggagtggcca a                                                          4271


<210>  6
<211>  145
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  5' ITR

<400>  6
ttggccactc cctctctgcg cgctcgctcg ctcactgagg ccgggcgacc aaaggtcgcc       60

cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc gagcgcgcag agagggagtg      120

gccaactcca tcactagggg ttcct                                            145


<210>  7
<211>  151
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  3' ITR

<400>  7
agccggagga acccctagtg atggagttgg ccactccctc tctgcgcgct cgctcgctca       60

ctgaggccgc ccgggcaaag cccgggcgtc gggcgacctt tggtcgcccg gcctcagtga      120

gcgagcgagc gcgcagagag ggagtggcca a                                     151


<210>  8
<211>  260
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Polyadenylation signal

<400>  8
gagctcgggg atccagacat gataagatac attgatgagt ttggacaaac cacaactaga       60

atgcagtgaa aaaaatgctt tatttgtgaa atttgtgatg ctattgcttt atttgtaacc      120

attataagct gcaataaaca agttaacaac aacaattgca ttcattttat gtttcaggtt      180

cagggggagg tgtgggaggt tttttaaagc aagtaaaacc tctacaaatg tggtatggct      240

gattatgatc aatgcatcct                                                  260


<210>  9
<211>  745
<212>  PRT
<213>  Artificial Sequence

<220>
<223>  variant AAV capsid protein

<400>  9

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


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


Lys Ala Ala Glu Arg His Lys Asp Asp Ser Arg Gly Leu Val Leu Pro 
        35                  40                  45              


Gly Tyr Lys Tyr Leu Gly Pro Phe Asn Gly Leu Asp Lys Gly Glu Pro 
    50                  55                  60                  


Val Asn Glu Ala Asp Ala Ala Ala Leu Glu His Asp Lys Ala Tyr Asp 
65                  70                  75                  80  


Arg Gln Leu Asp Ser Gly Asp Asn Pro Tyr Leu Lys Tyr Asn His Ala 
                85                  90                  95      


Asp Ala Glu Phe Gln Glu Arg Leu Lys Glu Asp Thr Ser Phe Gly Gly 
            100                 105                 110         


Asn Leu Gly Arg Ala Val Phe Gln Ala Lys Lys Arg Val Leu Glu Pro 
        115                 120                 125             


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


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


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


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


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


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


Ser Gly Asn Trp His Cys Asp Ser Thr Trp Met Gly Asp Arg Val Ile 
225                 230                 235                 240 


Thr Thr Ser Thr Arg Thr Trp Ala Leu Pro Thr Tyr Asn Asn His Leu 
                245                 250                 255     


Tyr Lys Gln Ile Ser Ser Gln Ser Gly Ala Ser Asn Asp Asn His Tyr 
            260                 265                 270         


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


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


Gly Ser Val Ser Thr Asn Leu Gln Arg Gly Asn Leu Ala Ile Ser Asp 
            580                 585                 590         


Gln Thr Lys His Ala Arg Gln Ala Ala Thr Ala Asp Val Asn Thr Gln 
        595                 600                 605             


Gly Val Leu Pro Gly Met Val Trp Gln Asp Arg Asp Val Tyr Leu Gln 
    610                 615                 620                 


Gly Pro Ile Trp Ala Lys Ile Pro His Thr Asp Gly His Phe His Pro 
625                 630                 635                 640 


Ser Pro Leu Met Gly Gly Phe Gly Leu Lys His Pro Pro Pro Gln Ile 
                645                 650                 655     


Leu Ile Lys Asn Thr Pro Val Pro Ala Asn Pro Ser Thr Thr Phe Ser 
            660                 665                 670         


Ala Ala Lys Phe Ala Ser Phe Ile Thr Gln Tyr Ser Thr Gly Gln Val 
        675                 680                 685             


Ser Val Glu Ile Glu Trp Glu Leu Gln Lys Glu Asn Ser Lys Arg Trp 
    690                 695                 700                 


Asn Pro Glu Ile Gln Tyr Thr Ser Asn Tyr Asn Lys Ser Val Asn Val 
705                 710                 715                 720 


Asp Phe Thr Val Asp Thr Asn Gly Val Tyr Ser Glu Pro Arg Pro Ile 
                725                 730                 735     


Gly Thr Arg Tyr Leu Thr Arg Asn Leu 
            740                 745 


