                         SEQUENCE LISTING

<110>  Prevail Therapeutics, Inc.
       Dai, Yong
       Zhou, Jingmin
       Daniels, Garrett
       Chan, Jonathan
       Haller, Jorge
       Nelson, Stuart
 
<120>  RECOMBINANT ADENO-ASSOCIATED VIRUS COMPOSITIONS AND METHODS FOR 
       PRODUCING SAME

<130>  PRVL-013/01WO 334806-2132

<150>  US 63/092,179
<151>  2020-10-15

<160>  12    

<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>  593
<212>  PRT
<213>  Homo sapiens

<400>  3

Met Trp Thr Leu Val Ser Trp Val Ala Leu Thr Ala Gly Leu Val Ala 
1               5                   10                  15      


Gly Thr Arg Cys Pro Asp Gly Gln Phe Cys Pro Val Ala Cys Cys Leu 
            20                  25                  30          


Asp Pro Gly Gly Ala Ser Tyr Ser Cys Cys Arg Pro Leu Leu Asp Lys 
        35                  40                  45              


Trp Pro Thr Thr Leu Ser Arg His Leu Gly Gly Pro Cys Gln Val Asp 
    50                  55                  60                  


Ala His Cys Ser Ala Gly His Ser Cys Ile Phe Thr Val Ser Gly Thr 
65                  70                  75                  80  


Ser Ser Cys Cys Pro Phe Pro Glu Ala Val Ala Cys Gly Asp Gly His 
                85                  90                  95      


His Cys Cys Pro Arg Gly Phe His Cys Ser Ala Asp Gly Arg Ser Cys 
            100                 105                 110         


Phe Gln Arg Ser Gly Asn Asn Ser Val Gly Ala Ile Gln Cys Pro Asp 
        115                 120                 125             


Ser Gln Phe Glu Cys Pro Asp Phe Ser Thr Cys Cys Val Met Val Asp 
    130                 135                 140                 


Gly Ser Trp Gly Cys Cys Pro Met Pro Gln Ala Ser Cys Cys Glu Asp 
145                 150                 155                 160 


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


Arg Cys Ile Thr Pro Thr Gly Thr His Pro Leu Ala Lys Lys Leu Pro 
            180                 185                 190         


Ala Gln Arg Thr Asn Arg Ala Val Ala Leu Ser Ser Ser Val Met Cys 
        195                 200                 205             


Pro Asp Ala Arg Ser Arg Cys Pro Asp Gly Ser Thr Cys Cys Glu Leu 
    210                 215                 220                 


Pro Ser Gly Lys Tyr Gly Cys Cys Pro Met Pro Asn Ala Thr Cys Cys 
225                 230                 235                 240 


Ser Asp His Leu His Cys Cys Pro Gln Asp Thr Val Cys Asp Leu Ile 
                245                 250                 255     


Gln Ser Lys Cys Leu Ser Lys Glu Asn Ala Thr Thr Asp Leu Leu Thr 
            260                 265                 270         


Lys Leu Pro Ala His Thr Val Gly Asp Val Lys Cys Asp Met Glu Val 
        275                 280                 285             


Ser Cys Pro Asp Gly Tyr Thr Cys Cys Arg Leu Gln Ser Gly Ala Trp 
    290                 295                 300                 


Gly Cys Cys Pro Phe Thr Gln Ala Val Cys Cys Glu Asp His Ile His 
305                 310                 315                 320 


Cys Cys Pro Ala Gly Phe Thr Cys Asp Thr Gln Lys Gly Thr Cys Glu 
                325                 330                 335     


Gln Gly Pro His Gln Val Pro Trp Met Glu Lys Ala Pro Ala His Leu 
            340                 345                 350         


Ser Leu Pro Asp Pro Gln Ala Leu Lys Arg Asp Val Pro Cys Asp Asn 
        355                 360                 365             


Val Ser Ser Cys Pro Ser Ser Asp Thr Cys Cys Gln Leu Thr Ser Gly 
    370                 375                 380                 


Glu Trp Gly Cys Cys Pro Ile Pro Glu Ala Val Cys Cys Ser Asp His 
385                 390                 395                 400 


Gln His Cys Cys Pro Gln Gly Tyr Thr Cys Val Ala Glu Gly Gln Cys 
                405                 410                 415     


Gln Arg Gly Ser Glu Ile Val Ala Gly Leu Glu Lys Met Pro Ala Arg 
            420                 425                 430         


Arg Ala Ser Leu Ser His Pro Arg Asp Ile Gly Cys Asp Gln His Thr 
        435                 440                 445             


Ser Cys Pro Val Gly Gln Thr Cys Cys Pro Ser Leu Gly Gly Ser Trp 
    450                 455                 460                 


Ala Cys Cys Gln Leu Pro His Ala Val Cys Cys Glu Asp Arg Gln His 
465                 470                 475                 480 


Cys Cys Pro Ala Gly Tyr Thr Cys Asn Val Lys Ala Arg Ser Cys Glu 
                485                 490                 495     


Lys Glu Val Val Ser Ala Gln Pro Ala Thr Phe Leu Ala Arg Ser Pro 
            500                 505                 510         


His Val Gly Val Lys Asp Val Glu Cys Gly Glu Gly His Phe Cys His 
        515                 520                 525             


Asp Asn Gln Thr Cys Cys Arg Asp Asn Arg Gln Gly Trp Ala Cys Cys 
    530                 535                 540                 


Pro Tyr Arg Gln Gly Val Cys Cys Ala Asp Arg Arg His Cys Cys Pro 
545                 550                 555                 560 


Ala Gly Phe Arg Cys Ala Ala Arg Gly Thr Lys Cys Leu Arg Arg Glu 
                565                 570                 575     


Ala Pro Arg Trp Asp Ala Pro Leu Arg Asp Pro Ala Leu Arg Gln Leu 
            580                 585                 590         


Leu 
    


<210>  4
<211>  1779
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  Codon-optimized Progranulin

<400>  4
atgtggaccc tggtgagctg ggtggccctg accgccggcc tggtggccgg cacccgctgc       60

cccgacggcc agttctgccc cgtggcctgc tgcctggacc ccggcggcgc cagctacagc      120

tgctgccgcc ccctgctgga caagtggccc accaccctga gccgccacct gggcggcccc      180

tgccaggtgg acgcccactg cagcgccggc cacagctgca tcttcaccgt gagcggcacc      240

agcagctgct gccccttccc cgaggccgtg gcctgcggcg acggccacca ctgctgcccc      300

cgcggcttcc actgcagcgc cgacggccgc agctgcttcc agcgcagcgg caacaacagc      360

gtgggcgcca tccagtgccc cgacagccag ttcgagtgcc ccgacttcag cacctgctgc      420

gtgatggtgg acggcagctg gggctgctgc cccatgcccc aggccagctg ctgcgaggac      480

cgcgtgcact gctgccccca cggcgccttc tgcgacctgg tgcacacccg ctgcatcacc      540

cccaccggca cccaccccct ggccaagaag ctgcccgccc agcgcaccaa ccgcgccgtg      600

gccctgagca gcagcgtgat gtgccccgac gcccgcagcc gctgccccga cggcagcacc      660

tgctgcgagc tgcccagcgg caagtacggc tgctgcccca tgcccaacgc cacctgctgc      720

agcgaccacc tgcactgctg cccccaggac accgtgtgcg acctgatcca gagcaagtgc      780

ctgagcaagg agaacgccac caccgacctg ctgaccaagc tgcccgccca caccgtgggc      840

gacgtgaagt gcgacatgga ggtgagctgc cccgacggct acacctgctg ccgcctgcag      900

agcggcgcct ggggctgctg ccccttcacc caggccgtgt gctgcgagga ccacatccac      960

tgctgccccg ccggcttcac ctgcgacacc cagaagggca cctgcgagca gggcccccac     1020

caggtgccct ggatggagaa ggcccccgcc cacctgagcc tgcccgaccc ccaggccctg     1080

aagcgcgacg tgccctgcga caacgtgagc agctgcccca gcagcgacac ctgctgccag     1140

ctgaccagcg gcgagtgggg ctgctgcccc atccccgagg ccgtgtgctg cagcgaccac     1200

cagcactgct gcccccaggg ctacacctgc gtggccgagg gccagtgcca gcgcggcagc     1260

gagatcgtgg ccggcctgga gaagatgccc gcccgccgcg ccagcctgag ccacccccgc     1320

gacatcggct gcgaccagca caccagctgc cccgtgggcc agacctgctg ccccagcctg     1380

ggcggcagct gggcctgctg ccagctgccc cacgccgtgt gctgcgagga ccgccagcac     1440

tgctgccccg ccggctacac ctgcaacgtg aaggcccgca gctgcgagaa ggaggtggtg     1500

agcgcccagc ccgccacctt cctggcccgc agcccccacg tgggcgtgaa ggacgtggag     1560

tgcggcgagg gccacttctg ccacgacaac cagacctgct gccgcgacaa ccgccagggc     1620

tgggcctgct gcccctaccg ccagggcgtg tgctgcgccg accgccgcca ctgctgcccc     1680

gccggcttcc gctgcgccgc ccgcggcacc aagtgcctgc gccgcgaggc cccccgctgg     1740

gacgcccccc tgcgcgaccc cgccctgcgc cagctgctg                            1779


<210>  5
<211>  145
<212>  DNA
<213>  Dependoparvovirus Adeno-associated virus 2

<400>  5
aggaacccct agtgatggag ttggccactc cctctctgcg cgctcgctcg ctcactgagg       60

ccgggcgacc aaaggtcgcc cgacgcccgg gctttgcccg ggcggcctca gtgagcgagc      120

gagcgcgcag agagggagtg gccaa                                            145


<210>  6
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAV9-GBA1 specific primer

<400>  6
gactgtggga tccgttcgaa                                                   20


<210>  7
<211>  19
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAV9-GBA1 specific primer

<400>  7
gattgacacc cggctcaga                                                    19


<210>  8
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAV9-GBA1 specific probe


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  6-FAM fluorescent dye attached

<220>
<221>  misc_feature
<222>  (23)..(23)
<223>  TAMRA fluorescent dye attached

<400>  8
ccatggaatt cagcagcccc agc                                               23


<210>  9
<211>  20
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAV9-GRN specific primer

<400>  9
gtcttccacg actgtgggat                                                   20


<210>  10
<211>  18
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAV9-GRN specific primer

<400>  10
gtcagggcca cccagctc                                                     18


<210>  11
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AAV9-GRN specific probe


<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  6-FAM fluorescent dye attached

<220>
<221>  misc_feature
<222>  (24)..(24)
<223>  TAMRA fluorescent dye attached

<400>  11
ccggttgagc caccatgtgg accc                                              24


<210>  12
<211>  23
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  inhibitory nucleic acid targeting alpha-synuclein

<400>  12
tggaagactt cgagatacac tgt                                               23


