                         SEQUENCE LISTING

<110>  THE CHILDREN'S HOSPITAL OF PHILADELPHIA
 
<120>  TREATMENT OF LYSOSOMAL STORAGE DISEASE IN THE EYE THROUGH 
       ADMINISTRATION OF AAVS EXPRESSING TPP1

<130>  CHOP.P0026WO

<140>  not yet known
<141>  2020-04-08

<150>  US 62/831,067
<151>  2019-04-08

<160>  2     

<170>  PatentIn version 3.5

<210>  1
<211>  563
<212>  PRT
<213>  Homo sapiens

<400>  1

Met Gly Leu Gln Ala Cys Leu Leu Gly Leu Phe Ala Leu Ile Leu Ser 
1               5                   10                  15      


Gly Lys Cys Ser Tyr Ser Pro Glu Pro Asp Gln Arg Arg Thr Leu Pro 
            20                  25                  30          


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


Leu Thr Phe Ala Leu Arg Gln Gln Asn Val Glu Arg Leu Ser Glu Leu 
    50                  55                  60                  


Val Gln Ala Val Ser Asp Pro Ser Ser Pro Gln Tyr Gly Lys Tyr Leu 
65                  70                  75                  80  


Thr Leu Glu Asn Val Ala Asp Leu Val Arg Pro Ser Pro Leu Thr Leu 
                85                  90                  95      


His Thr Val Gln Lys Trp Leu Leu Ala Ala Gly Ala Gln Lys Cys His 
            100                 105                 110         


Ser Val Ile Thr Gln Asp Phe Leu Thr Cys Trp Leu Ser Ile Arg Gln 
        115                 120                 125             


Ala Glu Leu Leu Leu Pro Gly Ala Glu Phe His His Tyr Val Gly Gly 
    130                 135                 140                 


Pro Thr Glu Thr His Val Val Arg Ser Pro His Pro Tyr Gln Leu Pro 
145                 150                 155                 160 


Gln Ala Leu Ala Pro His Val Asp Phe Val Gly Gly Leu His Arg Phe 
                165                 170                 175     


Pro Pro Thr Ser Ser Leu Arg Gln Arg Pro Glu Pro Gln Val Thr Gly 
            180                 185                 190         


Thr Val Gly Leu His Leu Gly Val Thr Pro Ser Val Ile Arg Lys Arg 
        195                 200                 205             


Tyr Asn Leu Thr Ser Gln Asp Val Gly Ser Gly Thr Ser Asn Asn Ser 
    210                 215                 220                 


Gln Ala Cys Ala Gln Phe Leu Glu Gln Tyr Phe His Asp Ser Asp Leu 
225                 230                 235                 240 


Ala Gln Phe Met Arg Leu Phe Gly Gly Asn Phe Ala His Gln Ala Ser 
                245                 250                 255     


Val Ala Arg Val Val Gly Gln Gln Gly Arg Gly Arg Ala Gly Ile Glu 
            260                 265                 270         


Ala Ser Leu Asp Val Gln Tyr Leu Met Ser Ala Gly Ala Asn Ile Ser 
        275                 280                 285             


Thr Trp Val Tyr Ser Ser Pro Gly Arg His Glu Gly Gln Glu Pro Phe 
    290                 295                 300                 


Leu Gln Trp Leu Met Leu Leu Ser Asn Glu Ser Ala Leu Pro His Val 
305                 310                 315                 320 


His Thr Val Ser Tyr Gly Asp Asp Glu Asp Ser Leu Ser Ser Ala Tyr 
                325                 330                 335     


Ile Gln Arg Val Asn Thr Glu Leu Met Lys Ala Ala Ala Arg Gly Leu 
            340                 345                 350         


Thr Leu Leu Phe Ala Ser Gly Asp Ser Gly Ala Gly Cys Trp Ser Val 
        355                 360                 365             


Ser Gly Arg His Gln Phe Arg Pro Thr Phe Pro Ala Ser Ser Pro Tyr 
    370                 375                 380                 


Val Thr Thr Val Gly Gly Thr Ser Phe Gln Glu Pro Phe Leu Ile Thr 
385                 390                 395                 400 


Asn Glu Ile Val Asp Tyr Ile Ser Gly Gly Gly Phe Ser Asn Val Phe 
                405                 410                 415     


Pro Arg Pro Ser Tyr Gln Glu Glu Ala Val Thr Lys Phe Leu Ser Ser 
            420                 425                 430         


Ser Pro His Leu Pro Pro Ser Ser Tyr Phe Asn Ala Ser Gly Arg Ala 
        435                 440                 445             


Tyr Pro Asp Val Ala Ala Leu Ser Asp Gly Tyr Trp Val Val Ser Asn 
    450                 455                 460                 


Arg Val Pro Ile Pro Trp Val Ser Gly Thr Ser Ala Ser Thr Pro Val 
465                 470                 475                 480 


Phe Gly Gly Ile Leu Ser Leu Ile Asn Glu His Arg Ile Leu Ser Gly 
                485                 490                 495     


Arg Pro Pro Leu Gly Phe Leu Asn Pro Arg Leu Tyr Gln Gln His Gly 
            500                 505                 510         


Ala Gly Leu Phe Asp Val Thr Arg Gly Cys His Glu Ser Cys Leu Asp 
        515                 520                 525             


Glu Glu Val Glu Gly Gln Gly Phe Cys Ser Gly Pro Gly Trp Asp Pro 
    530                 535                 540                 


Val Thr Gly Trp Gly Thr Pro Asn Phe Pro Ala Leu Leu Lys Thr Leu 
545                 550                 555                 560 


Leu Asn Pro 
            


<210>  2
<211>  1692
<212>  DNA
<213>  Homo sapiens

<400>  2
atgggactcc aagcctgcct cctagggctc tttgccctca tcctctctgg caaatgcagt       60

tacagcccgg agcccgacca gcggaggacg ctgcccccag gctgggtgtc cctgggccgt      120

gcggaccctg aggaagagct gagtctcacc tttgccctga gacagcagaa tgtggaaaga      180

ctctcggagc tggtgcaggc tgtgtcggat cccagctctc ctcaatacgg aaaatacctg      240

accctagaga atgtggctga tctggtgagg ccatccccac tgaccctcca cacggtgcaa      300

aaatggctct tggcagccgg agcccagaag tgccattctg tgatcacaca ggactttctg      360

acttgctggc tgagcatccg acaagcagag ctgctgctcc ctggggctga gtttcatcac      420

tatgtgggag gacctacgga aacccatgtt gtaaggtccc cacatcccta ccagcttcca      480

caggccttgg ccccccatgt ggactttgtg gggggactgc accgttttcc cccaacatca      540

tccctgaggc aacgtcctga gccgcaggtg acagggactg taggcctgca tctgggggta      600

accccctctg tgatccgtaa gcgatacaac ttgacctcac aagacgtggg ctctggcacc      660

agcaataaca gccaagcctg tgcccagttc ctggagcagt atttccatga ctcagacctg      720

gctcagttca tgcgcctctt cggtggcaac tttgcacatc aggcatcagt agcccgtgtg      780

gttggacaac agggccgggg ccgggccggg attgaggcca gtctagatgt gcagtacctg      840

atgagtgctg gtgccaacat ctccacctgg gtctacagta gccctggccg gcatgaggga      900

caggagccct tcctgcagtg gctcatgctg ctcagtaatg agtcagccct gccacatgtg      960

catactgtga gctatggaga tgatgaggac tccctcagca gcgcctacat ccagcgggtc     1020

aacactgagc tcatgaaggc tgccgctcgg ggtctcaccc tgctcttcgc ctcaggtgac     1080

agtggggccg ggtgttggtc tgtctctgga agacaccagt tccgccctac cttccctgcc     1140

tccagcccct atgtcaccac agtgggaggc acatccttcc aggaaccttt cctcatcaca     1200

aatgaaattg ttgactatat cagtggtggt ggcttcagca atgtgttccc acggccttca     1260

taccaggagg aagctgtaac gaagttcctg agctctagcc cccacctgcc accatccagt     1320

tacttcaatg ccagtggccg tgcctaccca gatgtggctg cactttctga tggctactgg     1380

gtggtcagca acagagtgcc cattccatgg gtgtccggaa cctcggcctc tactccagtg     1440

tttgggggga tcctatcctt gatcaatgag cacaggatcc ttagtggccg cccccctctt     1500

ggctttctca acccaaggct ctaccagcag catggggcag gactctttga tgtaacccgt     1560

ggctgccatg agtcctgtct ggatgaagag gtagagggcc agggtttctg ctctggtcct     1620

ggctgggatc ctgtaacagg ctggggaaca cccaacttcc cagctttgct gaagactcta     1680

ctcaacccct ga                                                         1692


