                         SEQUENCE LISTING

<110>  Prevail Therapeutics, Inc.
       NG, Mary 
 
<120>  ASSAY FOR MEASURING POTENCY OF GENE THERAPY DRUG PRODUCT

<130>  PRVL-017/01WO 334806-2133

<150>  US 63/092,189
<151>  2020-10-15

<160>  3     

<170>  PatentIn version 3.5

<210>  1
<211>  536
<212>  PRT
<213>  Homo sapiens

<400>  1

Met Glu Phe Ser Ser Pro Ser Arg Glu Glu Cys Pro Lys Pro Leu Ser 
1               5                   10                  15      


Arg Val Ser Ile Met Ala Gly Ser Leu Thr Gly Leu Leu Leu Leu Gln 
            20                  25                  30          


Ala Val Ser Trp Ala Ser Gly Ala Arg Pro Cys Ile Pro Lys Ser Phe 
        35                  40                  45              


Gly Tyr Ser Ser Val Val Cys Val Cys Asn Ala Thr Tyr Cys Asp Ser 
    50                  55                  60                  


Phe Asp Pro Pro Thr Phe Pro Ala Leu Gly Thr Phe Ser Arg Tyr Glu 
65                  70                  75                  80  


Ser Thr Arg Ser Gly Arg Arg Met Glu Leu Ser Met Gly Pro Ile Gln 
                85                  90                  95      


Ala Asn His Thr Gly Thr Gly Leu Leu Leu Thr Leu Gln Pro Glu Gln 
            100                 105                 110         


Lys Phe Gln Lys Val Lys Gly Phe Gly Gly Ala Met Thr Asp Ala Ala 
        115                 120                 125             


Ala Leu Asn Ile Leu Ala Leu Ser Pro Pro Ala Gln Asn Leu Leu Leu 
    130                 135                 140                 


Lys Ser Tyr Phe Ser Glu Glu Gly Ile Gly Tyr Asn Ile Ile Arg Val 
145                 150                 155                 160 


Pro Met Ala Ser Cys Asp Phe Ser Ile Arg Thr Tyr Thr Tyr Ala Asp 
                165                 170                 175     


Thr Pro Asp Asp Phe Gln Leu His Asn Phe Ser Leu Pro Glu Glu Asp 
            180                 185                 190         


Thr Lys Leu Lys Ile Pro Leu Ile His Arg Ala Leu Gln Leu Ala Gln 
        195                 200                 205             


Arg Pro Val Ser Leu Leu Ala Ser Pro Trp Thr Ser Pro Thr Trp Leu 
    210                 215                 220                 


Lys Thr Asn Gly Ala Val Asn Gly Lys Gly Ser Leu Lys Gly Gln Pro 
225                 230                 235                 240 


Gly Asp Ile Tyr His Gln Thr Trp Ala Arg Tyr Phe Val Lys Phe Leu 
                245                 250                 255     


Asp Ala Tyr Ala Glu His Lys Leu Gln Phe Trp Ala Val Thr Ala Glu 
            260                 265                 270         


Asn Glu Pro Ser Ala Gly Leu Leu Ser Gly Tyr Pro Phe Gln Cys Leu 
        275                 280                 285             


Gly Phe Thr Pro Glu His Gln Arg Asp Phe Ile Ala Arg Asp Leu Gly 
    290                 295                 300                 


Pro Thr Leu Ala Asn Ser Thr His His Asn Val Arg Leu Leu Met Leu 
305                 310                 315                 320 


Asp Asp Gln Arg Leu Leu Leu Pro His Trp Ala Lys Val Val Leu Thr 
                325                 330                 335     


Asp Pro Glu Ala Ala Lys Tyr Val His Gly Ile Ala Val His Trp Tyr 
            340                 345                 350         


Leu Asp Phe Leu Ala Pro Ala Lys Ala Thr Leu Gly Glu Thr His Arg 
        355                 360                 365             


Leu Phe Pro Asn Thr Met Leu Phe Ala Ser Glu Ala Cys Val Gly Ser 
    370                 375                 380                 


Lys Phe Trp Glu Gln Ser Val Arg Leu Gly Ser Trp Asp Arg Gly Met 
385                 390                 395                 400 


Gln Tyr Ser His Ser Ile Ile Thr Asn Leu Leu Tyr His Val Val Gly 
                405                 410                 415     


Trp Thr Asp Trp Asn Leu Ala Leu Asn Pro Glu Gly Gly Pro Asn Trp 
            420                 425                 430         


Val Arg Asn Phe Val Asp Ser Pro Ile Ile Val Asp Ile Thr Lys Asp 
        435                 440                 445             


Thr Phe Tyr Lys Gln Pro Met Phe Tyr His Leu Gly His Phe Ser Lys 
    450                 455                 460                 


Phe Ile Pro Glu Gly Ser Gln Arg Val Gly Leu Val Ala Ser Gln Lys 
465                 470                 475                 480 


Asn Asp Leu Asp Ala Val Ala Leu Met His Pro Asp Gly Ser Ala Val 
                485                 490                 495     


Val Val Val Leu Asn Arg Ser Ser Lys Asp Val Pro Leu Thr Ile Lys 
            500                 505                 510         


Asp Pro Ala Val Gly Phe Leu Glu Thr Ile Ser Pro Gly Tyr Ser Ile 
        515                 520                 525             


His Thr Tyr Leu Trp Arg Arg Gln 
    530                 535     


<210>  2
<211>  1608
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Codon-optimized Glucocerebrosidase

<400>  2
atggaattca gcagccccag cagagaggaa tgccccaagc ctctgagccg ggtgtcaatc       60

atggccggat ctctgacagg actgctgctg cttcaggccg tgtcttgggc ttctggcgct      120

agaccttgca tccccaagag cttcggctac agcagcgtcg tgtgcgtgtg caatgccacc      180

tactgcgaca gcttcgaccc tcctaccttt cctgctctgg gcaccttcag cagatacgag      240

agcaccagat ccggcagacg gatggaactg agcatgggac ccatccaggc caatcacaca      300

ggcactggcc tgctgctgac actgcagcct gagcagaaat tccagaaagt gaaaggcttc      360

ggcggagcca tgacagatgc cgccgctctg aatatcctgg ctctgtctcc accagctcag      420

aacctgctgc tcaagagcta cttcagcgag gaaggcatcg gctacaacat catcagagtg      480

cccatggcca gctgcgactt cagcatcagg acctacacct acgccgacac acccgacgat      540

ttccagctgc acaacttcag cctgcctgaa gaggacacca agctgaagat ccctctgatc      600

cacagagccc tgcagctggc acaaagaccc gtgtcactgc tggcctctcc atggacatct      660

cccacctggc tgaaaacaaa tggcgccgtg aatggcaagg gcagcctgaa aggccaacct      720

ggcgacatct accaccagac ctgggccaga tacttcgtga agttcctgga cgcctatgcc      780

gagcacaagc tgcagttttg ggccgtgaca gccgagaacg aaccttctgc tggactgctg      840

agcggctacc cctttcagtg cctgggcttt acacccgagc accagcggga ctttatcgcc      900

cgtgatctgg gacccacact ggccaatagc acccaccata atgtgcggct gctgatgctg      960

gacgaccaga gactgcttct gccccactgg gctaaagtgg tgctgacaga tcctgaggcc     1020

gccaaatacg tgcacggaat cgccgtgcac tggtatctgg actttctggc ccctgccaag     1080

gccacactgg gagagacaca cagactgttc cccaacacca tgctgttcgc cagcgaagcc     1140

tgtgtgggca gcaagttttg ggaacagagc gtgcggctcg gcagctggga tagaggcatg     1200

cagtacagcc acagcatcat caccaacctg ctgtaccacg tcgtcggctg gaccgactgg     1260

aatctggccc tgaatcctga aggcggccct aactgggtcc gaaacttcgt ggacagcccc     1320

atcatcgtgg acatcaccaa ggacaccttc tacaagcagc ccatgttcta ccacctggga     1380

cacttcagca agttcatccc cgagggctct cagcgcgttg gactggtggc ttcccagaag     1440

aacgatctgg acgccgtggc tctgatgcac cctgatggat ctgctgtggt ggtggtcctg     1500

aaccgcagca gcaaagatgt gcccctgacc atcaaggatc ccgccgtggg attcctggaa     1560

acaatcagcc ctggctactc catccacacc tacctgtggc gtagacag                  1608


<210>  3
<211>  145
<212>  DNA
<213>  Dependoparvovirus Adeno-associated virus 2

<400>  3
aggaacccct agtgatggag ttggccactc cctctctgcg cgctcgctcg ctcactgagg       60

ccgggcgacc aaaggtcgcc cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc      120

gagcgcgcag agagggagtg gccaa                                            145


