                         SEQUENCE LISTING

<110>  Research Institute at Nationwide Children's Hospital
       Sanford Research
 
<120>  OPTIMIZED GENE THERAPY TARGETING RETINAL CELLS 

<130>  28335/53953

<150>  US 62/849,794
<151>  2019-05-17

<160>  9     

<170>  PatentIn version 3.5

<210>  1
<211>  1317
<212>  DNA
<213>  Homo sapiens

<400>  1
atgggaggct gtgcaggctc gcggcggcgc ttttcggatt ccgaggggga ggagaccgtc       60

ccggagcccc ggctccctct gttggaccat cagggcgcgc attggaagaa cgcggtgggc      120

ttctggctgc tgggcctttg caacaacttc tcttatgtgg tgatgctgag tgccgcccac      180

gacatcctta gccacaagag gacatcggga aaccagagcc atgtggaccc aggcccaacg      240

ccgatccccc acaacagctc atcacgattt gactgcaact ctgtctctac ggctgctgtg      300

ctcctggcgg acatcctccc cacactcgtc atcaaattgt tggctcctct tggccttcac      360

ctgctgccct acagcccccg ggttctcgtc agtgggattt gtgctgctgg aagcttcgtc      420

ctggttgcct tttctcattc tgtggggacc agcctgtgtg gtgtggtctt cgctagcatc      480

tcatcaggcc ttggggaggt caccttcctc tccctcactg ccttctaccc cagggccgtg      540

atctcctggt ggtcctcagg gactggggga gctgggctgc tgggggccct gtcctacctg      600

ggcctcaccc aggccggcct ctcccctcag cagaccctgc tgtccatgct gggtatccct      660

gccctgctgc tggccagcta tttcttgttg ctcacatctc ctgaggccca ggaccctgga      720

ggggaagaag aagcagagag cgcagcccgg cagcccctca taagaaccga ggccccggag      780

tcgaagccag gctccagctc cagcctctcc cttcgggaaa ggtggacagt gttcaagggt      840

ctgctgtggt acattgttcc cttggtcgta gtttactttg ccgagtattt cattaaccag      900

ggactttttg aactcctctt tttctggaac acttccctga gtcacgctca gcaataccgc      960

tggtaccaga tgctgtacca ggctggcgtc tttgcctccc gctcttctct ccgctgctgt     1020

cgcatccgtt tcacctgggc cctggccctg ctgcagtgcc tcaacctggt gttcctgctg     1080

gcagacgtgt ggttcggctt tctgccaagc atctacctcg tcttcctgat cattctgtat     1140

gaggggctcc tgggaggcgc agcctacgtg aacaccttcc acaacatcgc cctggagacc     1200

agtgatgagc accgggagtt tgcaatggcg gccacctgca tctctgacac actggggatc     1260

tccctgtcgg ggctcctggc tttgcctctg catgacttcc tctgccagct ctcctga        1317


<210>  2
<211>  438
<212>  PRT
<213>  Homo sapiens

<400>  2

Met Gly Gly Cys Ala Gly Ser Arg Arg Arg Phe Ser Asp Ser Glu Gly 
1               5                   10                  15      


Glu Glu Thr Val Pro Glu Pro Arg Leu Pro Leu Leu Asp His Gln Gly 
            20                  25                  30          


Ala His Trp Lys Asn Ala Val Gly Phe Trp Leu Leu Gly Leu Cys Asn 
        35                  40                  45              


Asn Phe Ser Tyr Val Val Met Leu Ser Ala Ala His Asp Ile Leu Ser 
    50                  55                  60                  


His Lys Arg Thr Ser Gly Asn Gln Ser His Val Asp Pro Gly Pro Thr 
65                  70                  75                  80  


Pro Ile Pro His Asn Ser Ser Ser Arg Phe Asp Cys Asn Ser Val Ser 
                85                  90                  95      


Thr Ala Ala Val Leu Leu Ala Asp Ile Leu Pro Thr Leu Val Ile Lys 
            100                 105                 110         


Leu Leu Ala Pro Leu Gly Leu His Leu Leu Pro Tyr Ser Pro Arg Val 
        115                 120                 125             


Leu Val Ser Gly Ile Cys Ala Ala Gly Ser Phe Val Leu Val Ala Phe 
    130                 135                 140                 


Ser His Ser Val Gly Thr Ser Leu Cys Gly Val Val Phe Ala Ser Ile 
145                 150                 155                 160 


Ser Ser Gly Leu Gly Glu Val Thr Phe Leu Ser Leu Thr Ala Phe Tyr 
                165                 170                 175     


Pro Arg Ala Val Ile Ser Trp Trp Ser Ser Gly Thr Gly Gly Ala Gly 
            180                 185                 190         


Leu Leu Gly Ala Leu Ser Tyr Leu Gly Leu Thr Gln Ala Gly Leu Ser 
        195                 200                 205             


Pro Gln Gln Thr Leu Leu Ser Met Leu Gly Ile Pro Ala Leu Leu Leu 
    210                 215                 220                 


Ala Ser Tyr Phe Leu Leu Leu Thr Ser Pro Glu Ala Gln Asp Pro Gly 
225                 230                 235                 240 


Gly Glu Glu Glu Ala Glu Ser Ala Ala Arg Gln Pro Leu Ile Arg Thr 
                245                 250                 255     


Glu Ala Pro Glu Ser Lys Pro Gly Ser Ser Ser Ser Leu Ser Leu Arg 
            260                 265                 270         


Glu Arg Trp Thr Val Phe Lys Gly Leu Leu Trp Tyr Ile Val Pro Leu 
        275                 280                 285             


Val Val Val Tyr Phe Ala Glu Tyr Phe Ile Asn Gln Gly Leu Phe Glu 
    290                 295                 300                 


Leu Leu Phe Phe Trp Asn Thr Ser Leu Ser His Ala Gln Gln Tyr Arg 
305                 310                 315                 320 


Trp Tyr Gln Met Leu Tyr Gln Ala Gly Val Phe Ala Ser Arg Ser Ser 
                325                 330                 335     


Leu Arg Cys Cys Arg Ile Arg Phe Thr Trp Ala Leu Ala Leu Leu Gln 
            340                 345                 350         


Cys Leu Asn Leu Val Phe Leu Leu Ala Asp Val Trp Phe Gly Phe Leu 
        355                 360                 365             


Pro Ser Ile Tyr Leu Val Phe Leu Ile Ile Leu Tyr Glu Gly Leu Leu 
    370                 375                 380                 


Gly Gly Ala Ala Tyr Val Asn Thr Phe His Asn Ile Ala Leu Glu Thr 
385                 390                 395                 400 


Ser Asp Glu His Arg Glu Phe Ala Met Ala Ala Thr Cys Ile Ser Asp 
                405                 410                 415     


Thr Leu Gly Ile Ser Leu Ser Gly Leu Leu Ala Leu Pro Leu His Asp 
            420                 425                 430         


Phe Leu Cys Gln Leu Ser 
        435             


<210>  3
<211>  936
<212>  DNA
<213>  Homo sapiens

<400>  3
atggaggcga cgcggaggcg gcagcacctg ggagcgacgg gcggcccagg cgcgcagctg       60

ggcgcctcct tcctgcaggc caggcatggc tctgtgagcg ctgatgaggc tgcccgcacg      120

gctcccttcc acctcgacct ctggttctac ttcacactgc agaactgggt tctggacttt      180

gggcgtccca ttgccatgct ggtattccct ctcgagtggt ttccactcaa caagcccagt      240

gttggggact acttccacat ggcctacaac gtcatcacgc cctttctctt gctcaagctc      300

atcgagcggt ccccccgcac cctgccacgc tccatcacgt acgtgagcat catcatcttc      360

atcatgggtg ccagcatcca cctggtgggt gactctgtca accaccgcct gctcttcagt      420

ggctaccagc accacctgtc tgtccgtgag aaccccatca tcaagaatct caagccggag      480

acgctgatcg actcctttga gctgctctac tattatgatg agtacctggg tcactgcatg      540

tggtacatcc ccttcttcct catcctcttc atgtacttca gcggctgctt tactgcctct      600

aaagctgaga gcttgattcc agggcctgcc ctgctcctgg tggcacccag tggcctgtac      660

tactggtacc tggtcaccga gggccagatc ttcatcctct tcatcttcac cttcttcgcc      720

atgctggccc tcgtcctgca ccagaagcgc aagcgcctct tcctggacag caacggcctc      780

ttcctcttct cctccttcgc actgaccctc ttgcttgtgg cgctctgggt cgcctggctg      840

tggaatgacc ctgttctcag gaagaagtac ccgggtgtca tctacgtccc tgagccctgg      900

gctttctaca cccttcacgt cagcagtcgg cactga                                936


<210>  4
<211>  311
<212>  PRT
<213>  Homo sapiens

<400>  4

Met Glu Ala Thr Arg Arg Arg Gln His Leu Gly Ala Thr Gly Gly Pro 
1               5                   10                  15      


Gly Ala Gln Leu Gly Ala Ser Phe Leu Gln Ala Arg His Gly Ser Val 
            20                  25                  30          


Ser Ala Asp Glu Ala Ala Arg Thr Ala Pro Phe His Leu Asp Leu Trp 
        35                  40                  45              


Phe Tyr Phe Thr Leu Gln Asn Trp Val Leu Asp Phe Gly Arg Pro Ile 
    50                  55                  60                  


Ala Met Leu Val Phe Pro Leu Glu Trp Phe Pro Leu Asn Lys Pro Ser 
65                  70                  75                  80  


Val Gly Asp Tyr Phe His Met Ala Tyr Asn Val Ile Thr Pro Phe Leu 
                85                  90                  95      


Leu Leu Lys Leu Ile Glu Arg Ser Pro Arg Thr Leu Pro Arg Ser Ile 
            100                 105                 110         


Thr Tyr Val Ser Ile Ile Ile Phe Ile Met Gly Ala Ser Ile His Leu 
        115                 120                 125             


Val Gly Asp Ser Val Asn His Arg Leu Leu Phe Ser Gly Tyr Gln His 
    130                 135                 140                 


His Leu Ser Val Arg Glu Asn Pro Ile Ile Lys Asn Leu Lys Pro Glu 
145                 150                 155                 160 


Thr Leu Ile Asp Ser Phe Glu Leu Leu Tyr Tyr Tyr Asp Glu Tyr Leu 
                165                 170                 175     


Gly His Cys Met Trp Tyr Ile Pro Phe Phe Leu Ile Leu Phe Met Tyr 
            180                 185                 190         


Phe Ser Gly Cys Phe Thr Ala Ser Lys Ala Glu Ser Leu Ile Pro Gly 
        195                 200                 205             


Pro Ala Leu Leu Leu Val Ala Pro Ser Gly Leu Tyr Tyr Trp Tyr Leu 
    210                 215                 220                 


Val Thr Glu Gly Gln Ile Phe Ile Leu Phe Ile Phe Thr Phe Phe Ala 
225                 230                 235                 240 


Met Leu Ala Leu Val Leu His Gln Lys Arg Lys Arg Leu Phe Leu Asp 
                245                 250                 255     


Ser Asn Gly Leu Phe Leu Phe Ser Ser Phe Ala Leu Thr Leu Leu Leu 
            260                 265                 270         


Val Ala Leu Trp Val Ala Trp Leu Trp Asn Asp Pro Val Leu Arg Lys 
        275                 280                 285             


Lys Tyr Pro Gly Val Ile Tyr Val Pro Glu Pro Trp Ala Phe Tyr Thr 
    290                 295                 300                 


Leu His Val Ser Ser Arg His 
305                 310     


<210>  5
<211>  861
<212>  DNA
<213>  Homo sapiens

<400>  5
atgaatcctg cgagcgatgg gggcacatca gagagcattt ttgacctgga ctatgcatcc       60

tgggggatcc gctccacgct gatggtcgct ggctttgtct tctacttggg cgtctttgtg      120

gtctgccacc agctgtcctc ttccctgaat gccacttacc gttctttggt ggccagagag      180

aaggtcttct gggacctggc ggccacgcgt gcagtctttg gtgttcagag cacagccgca      240

ggcctgtggg ctctgctggg ggaccctgtg ctgcatgccg acaaggcgcg tggccagcag      300

aactggtgct ggtttcacat cacgacagca acgggattct tttgctttga aaatgttgca      360

gtccacctgt ccaacttgat cttccggaca tttgacttgt ttctggttat ccaccatctc      420

tttgcctttc ttgggtttct tggctgcttg gtcaatctcc aagctggcca ctatctagct      480

atgaccacgt tgctcctgga gatgagcacg ccctttacct gcgtttcctg gatgctctta      540

aaggcgggct ggtccgagtc tctgttttgg aagctcaacc agtggctgat gattcacatg      600

tttcactgcc gcatggttct aacctaccac atgtggtggg tgtgtttctg gcactgggac      660

ggcctggtca gcagcctgta tctgcctcat ttgacactgt tccttgtcgg actggctctg      720

cttacgctaa tcattaatcc atattggacc cataagaaga ctcagcagct tctcaatccg      780

gtggactgga acttcgcaca gccagaagcc aagagcaggc cagaaggcaa cgggcagctg      840

ctgcggaaga agaggccata g                                                861


<210>  6
<211>  286
<212>  PRT
<213>  Homo sapiens

<400>  6

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


Asp Tyr Ala Ser Trp Gly Ile Arg Ser Thr Leu Met Val Ala Gly Phe 
            20                  25                  30          


Val Phe Tyr Leu Gly Val Phe Val Val Cys His Gln Leu Ser Ser Ser 
        35                  40                  45              


Leu Asn Ala Thr Tyr Arg Ser Leu Val Ala Arg Glu Lys Val Phe Trp 
    50                  55                  60                  


Asp Leu Ala Ala Thr Arg Ala Val Phe Gly Val Gln Ser Thr Ala Ala 
65                  70                  75                  80  


Gly Leu Trp Ala Leu Leu Gly Asp Pro Val Leu His Ala Asp Lys Ala 
                85                  90                  95      


Arg Gly Gln Gln Asn Trp Cys Trp Phe His Ile Thr Thr Ala Thr Gly 
            100                 105                 110         


Phe Phe Cys Phe Glu Asn Val Ala Val His Leu Ser Asn Leu Ile Phe 
        115                 120                 125             


Arg Thr Phe Asp Leu Phe Leu Val Ile His His Leu Phe Ala Phe Leu 
    130                 135                 140                 


Gly Phe Leu Gly Cys Leu Val Asn Leu Gln Ala Gly His Tyr Leu Ala 
145                 150                 155                 160 


Met Thr Thr Leu Leu Leu Glu Met Ser Thr Pro Phe Thr Cys Val Ser 
                165                 170                 175     


Trp Met Leu Leu Lys Ala Gly Trp Ser Glu Ser Leu Phe Trp Lys Leu 
            180                 185                 190         


Asn Gln Trp Leu Met Ile His Met Phe His Cys Arg Met Val Leu Thr 
        195                 200                 205             


Tyr His Met Trp Trp Val Cys Phe Trp His Trp Asp Gly Leu Val Ser 
    210                 215                 220                 


Ser Leu Tyr Leu Pro His Leu Thr Leu Phe Leu Val Gly Leu Ala Leu 
225                 230                 235                 240 


Leu Thr Leu Ile Ile Asn Pro Tyr Trp Thr His Lys Lys Thr Gln Gln 
                245                 250                 255     


Leu Leu Asn Pro Val Asp Trp Asn Phe Ala Gln Pro Glu Ala Lys Ser 
            260                 265                 270         


Arg Pro Glu Gly Asn Gly Gln Leu Leu Arg Lys Lys Arg Pro 
        275                 280                 285     


<210>  7
<211>  270
<212>  DNA
<213>  Homo sapiens

<400>  7
ccacgttctg cttcactctc cccatctccc ccccctcccc acccccaatt ttgtatttat       60

ttatttttta attattttgt gcagcgatgg gggcgggggg gggggggggg cgcgcgccag      120

gcggggcggg gcggggcgag gggcggggcg gggcgaggcg gagaggtgcg gcggcagcca      180

atcagagcgg cgcgctccga aagtttcctt ttatggcgag gcggcggcgg cggcggccct      240

ataaaaagcg aagcgcgcgg cgggcgggag                                       270


<210>  8
<211>  280
<212>  DNA
<213>  Homo sapiens

<400>  8
cgttacataa cttacggtaa atggcccgcc tggctgaccg cccaacgacc cccgcccatt       60

gacgtcaata atgacgtatg ttcccatagt aacgccaata gggactttcc attgacgtca      120

atgggtggag tatttacggt aaactgccca cttggcagta catcaagtgt atcatatgcc      180

aagtacgccc cctattgacg tcaatgacgg taaatggccc gcctggcatt atgcccagta      240

catgacctta tgggactttc ctacttggca gtacatctac                            280


<210>  9
<211>  546
<212>  DNA
<213>  Homo sapiens

<400>  9
gaacaacgcc aggctcctca acaggcaact ttgctacttc tacagaaaat gataataaag       60

aaatgctggt gaagtcaaat gcttatcaca atggtgaact actcagcagg gaggctctaa      120

taggcgccaa gagcctagac ttccttaagc gccagagtcc acaagggccc agttaatcct      180

caacattcaa atgctgccca caaaaccagc ccctctgtgc cctagccgcc tcttttttcc      240

aagtgacagt agaactccac caatccgcag ctgaatgggg tccgcctctt ttccctgcct      300

aaacagacag gaactcctgc caattgaggg cgtcaccgct aaggctccgc cccagcctgg      360

gctccacaac caatgaaggg taatctcgac aaagagcaag gggtggggcg cgggcgcgca      420

ggtgcagcag cacacaggct ggtcgggagg gcggggcgcg acgtctgccg tgcggggtcc      480

cggcatcggt tgcgcgcgcg ctccctcctc tcggagagag ggctgtggta aaacccgtcc      540

ggaaaa                                                                 546


