                         SEQUENCE LISTING

<110>  The Children's Hospital of Philadelphia
       HIGH, Katherine A.
       MINGOZZI, Federico
       SUN, Junwei
       JOHNSON, Philip
 
<120>  AAV VECTOR COMPOSITIONS AND METHODS FOR GENE TRANSFER TO CELLS, 
       ORGANS AND TISSUES

<130>  074659-0420998

<140>  PCT/US2013/26695
<141>  2013-02-19

<150>  61/600,415
<151>  2012-02-17

<160>  4     

<170>  PatentIn version 3.5

<210>  1
<211>  738
<212>  PRT
<213>  Adeno-associated virus

<400>  1

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


Glu Gly Ile Arg Glu Trp Trp Asp Leu Lys Pro Gly Ala Pro Lys Pro 
            20                  25                  30          


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


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


Asp Ala Glu Phe Gln Glu Arg Leu Gln 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 Ser Pro Val Lys Thr Ala Pro Gly Lys Lys Arg 
    130                 135                 140                 


Pro Val Glu Pro Ser Pro Gln Arg Ser Pro Asp Ser Ser Thr Gly Ile 
145                 150                 155                 160 


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


Thr Gly Asp Ser Glu Ser Val Pro Asp Pro Gln Pro Ile Gly Glu Pro 
            180                 185                 190         


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


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


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


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


Leu Tyr Lys Gln Ile Ser Asn Gly Thr Ser Gly Gly Ser Thr Asn Asp 
            260                 265                 270         


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


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


Asn Asn Trp Gly Phe Arg Pro Lys Arg Leu Asn Phe Lys Leu Phe Asn 
305                 310                 315                 320 


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


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


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


Pro Ala Asp Val Phe Met Ile Pro Gln Tyr Gly Tyr Leu Thr Leu Asn 
    370                 375                 380                 


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


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


Asn Phe Glu Asp Val Pro Phe His Ser Ser Tyr Ala His Ser Gln Ser 
            420                 425                 430         


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


Ser Arg Thr Gln Ser Thr Gly Gly Thr Ala Gly Thr Gln Gln Leu Leu 
    450                 455                 460                 


Phe Ser Gln Ala Gly Pro Asn Asn Met Ser Ala Gln Ala Lys Asn Trp 
465                 470                 475                 480 


Leu Pro Gly Pro Cys Tyr Arg Gln Gln Arg Val Ser Thr Thr Leu Ser 
                485                 490                 495     


Gln Asn Asn Asn Ser Asn Phe Ala Trp Thr Gly Ala Thr Lys Tyr His 
            500                 505                 510         


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


His Lys Asp Asp Glu Glu Arg Phe Phe Pro Ser Ser Gly Val Leu Met 
    530                 535                 540                 


Phe Gly Lys Gln Gly Ala Gly Lys Asp Asn Val Asp Tyr Ser Ser Val 
545                 550                 555                 560 


Met Leu Thr Ser Glu Glu Glu Ile Lys Thr Thr Asn Pro Val Ala Thr 
                565                 570                 575     


Glu Gln Tyr Gly Val Val Ala Asp Asn Leu Gln Gln Gln Asn Ala Ala 
            580                 585                 590         


Pro Ile Val Gly Ala Val Asn Ser Gln Gly Ala Leu Pro Gly Met Val 
        595                 600                 605             


Trp Gln Asn Arg Asp Val Tyr Leu Gln Gly Pro Ile Trp Ala Lys Ile 
    610                 615                 620                 


Pro His Thr Asp Gly Asn Phe His Pro Ser Pro Leu Met Gly Gly Phe 
625                 630                 635                 640 


Gly Leu Lys His Pro Pro Pro Gln Ile Leu Ile Lys Asn Thr Pro Val 
                645                 650                 655     


Pro Ala Asp Pro Pro Thr Thr Phe Asn Gln Ala Lys Leu Ala Ser Phe 
            660                 665                 670         


Ile Thr Gln Tyr Ser Thr Gly Gln Val Ser Val Glu Ile Glu Trp Glu 
        675                 680                 685             


Leu Gln Lys Glu Asn Ser Lys Arg Trp Asn Pro Glu Ile Gln Tyr Thr 
    690                 695                 700                 


Ser Asn Tyr Tyr Lys Ser Thr Asn Val Asp Phe Ala Val Asn Thr Glu 
705                 710                 715                 720 


Gly Thr Tyr Ser Glu Pro Arg Pro Ile Gly Thr Arg Tyr Leu Thr Arg 
                725                 730                 735     


Asn Leu 
        


<210>  2
<211>  2217
<212>  DNA
<213>  Adeno-associated virus

<400>  2
atggctgccg atggttatct tccagattgg ctcgaggaca acctctctga gggcattcgc       60

gagtggtggg acctgaaacc tggagccccg aaacccaaag ccaaccagca aaagcaggac      120

aacggccggg gtctggtgct tcctggctac aagtacctcg gacccttcaa cggactcgac      180

aagggggagc ccgtcaacgc ggcggacgca gcggccctcg agcacgacaa ggcctacgac      240

cagcagctcc aagcgggtga caatccgtac ctgcggtata atcacgccga cgccgagttt      300

caggagcgtc tgcaagaaga tacgtctttt gggggcaacc tcgggcgcgc agtcttccag      360

gccaaaaagc gggttctcga acctctgggc ctggttgaat cgccggttaa gacggctcct      420

ggaaagaaga gaccggtaga gccatcaccc cagcgctctc cagactcctc tacgggcatc      480

ggcaagaaag gccagcagcc cgcaaaaaag agactcaatt ttgggcagac tggcgactca      540

gagtcagtcc ccgaccctca accaatcgga gaaccaccag caggcccctc tggtctggga      600

tctggtacaa tggctgcagg cggtggcgct ccaatggcag acaataacga aggcgccgac      660

ggagtgggta gttcctcagg aaattggcat tgcgattcca catggctggg cgacagagtc      720

atcaccacca gcacccgcac ctgggccctg cccacctaca acaaccacct ctacaagcaa      780

atctccaacg ggacctcggg aggaagcacc aacgacaaca cctacttcgg ctacagcacc      840

ccctgggggt attttgactt caacagattc cactgccact tttcaccacg tgactggcag      900

cgactcatca acaacaactg gggattccgg cccaagaggc tcaacttcaa gctcttcaac      960

atccaagtca aggaggtcac gcagaatgaa ggcaccaaga ccatcgccaa taaccttacc     1020

agcacgattc aggtctttac ggactcggaa taccagctcc cgtacgtgct cggctcggcg     1080

caccagggct gcctgcctcc gttcccggcg gacgtcttca tgattcctca gtacgggtac     1140

ctgactctga acaatggcag tcaggctgtg ggccggtcgt ccttctactg cctggagtac     1200

tttccttctc aaatgctgag aacgggcaac aactttgaat tcagctacaa cttcgaggac     1260

gtgcccttcc acagcagcta cgcgcacagc cagagcctgg accggctgat gaaccctctc     1320

atcgaccagt acttgtacta cctgtcccgg actcaaagca cgggcggtac tgcaggaact     1380

cagcagttgc tattttctca ggccgggcct aacaacatgt cggctcaggc caagaactgg     1440

ctacccggtc cctgctaccg gcagcaacgc gtctccacga cactgtcgca gaacaacaac     1500

agcaactttg cctggacggg tgccaccaag tatcatctga atggcagaga ctctctggtg     1560

aatcctggcg ttgccatggc tacccacaag gacgacgaag agcgattttt tccatccagc     1620

ggagtcttaa tgtttgggaa acagggagct ggaaaagaca acgtggacta tagcagcgtg     1680

atgctaacca gcgaggaaga aataaagacc accaacccag tggccacaga acagtacggc     1740

gtggtggccg ataacctgca acagcaaaac gccgctccta ttgtaggggc cgtcaatagt     1800

caaggagcct tacctggcat ggtgtggcag aaccgggacg tgtacctgca gggtcccatc     1860

tgggccaaga ttcctcatac ggacggcaac tttcatccct cgccgctgat gggaggcttt     1920

ggactgaagc atccgcctcc tcagatcctg attaaaaaca cacctgttcc cgcggatcct     1980

ccgaccacct tcaatcaggc caagctggct tctttcatca cgcagtacag taccggccag     2040

gtcagcgtgg agatcgagtg ggagctgcag aaggagaaca gcaaacgctg gaacccagag     2100

attcagtaca cttccaacta ctacaaatct acaaatgtgg actttgctgt caatactgag     2160

ggtacttatt ccgagcctcg ccccattggc acccgttacc tcacccgtaa tctgtaa        2217


<210>  3
<211>  601
<212>  PRT
<213>  Adeno-associated virus

<400>  3

Thr Ala Pro Gly Lys Lys Arg Pro Val Glu Pro Ser Pro Gln Arg Ser 
1               5                   10                  15      


Pro Asp Ser Ser Thr Gly Ile Gly Lys Lys Gly Gln Gln Pro Ala Lys 
            20                  25                  30          


Lys Arg Leu Asn Phe Gly Gln Thr Gly Asp Ser Glu Ser Val Pro Asp 
        35                  40                  45              


Pro Gln Pro Ile Gly Glu Pro Pro Ala Gly Pro Ser Gly Leu Gly Ser 
    50                  55                  60                  


Gly Thr Met Ala Ala Gly Gly Gly Ala Pro Met Ala Asp Asn Asn Glu 
65                  70                  75                  80  


Gly Ala Asp Gly Val Gly Ser Ser Ser Gly Asn Trp His Cys Asp Ser 
                85                  90                  95      


Thr Trp Leu Gly Asp Arg Val Ile Thr Thr Ser Thr Arg Thr Trp Ala 
            100                 105                 110         


Leu Pro Thr Tyr Asn Asn His Leu Tyr Lys Gln Ile Ser Asn Gly Thr 
        115                 120                 125             


Ser Gly Gly Ser Thr Asn Asp Asn Thr Tyr Phe Gly Tyr Ser Thr Pro 
    130                 135                 140                 


Trp Gly Tyr Phe Asp Phe Asn Arg Phe His Cys His Phe Ser Pro Arg 
145                 150                 155                 160 


Asp Trp Gln Arg Leu Ile Asn Asn Asn Trp Gly Phe Arg Pro Lys Arg 
                165                 170                 175     


Leu Asn Phe Lys Leu Phe Asn Ile Gln Val Lys Glu Val Thr Gln Asn 
            180                 185                 190         


Glu Gly Thr Lys Thr Ile Ala Asn Asn Leu Thr Ser Thr Ile Gln Val 
        195                 200                 205             


Phe Thr Asp Ser Glu Tyr Gln Leu Pro Tyr Val Leu Gly Ser Ala His 
    210                 215                 220                 


Gln Gly Cys Leu Pro Pro Phe Pro Ala Asp Val Phe Met Ile Pro Gln 
225                 230                 235                 240 


Tyr Gly Tyr Leu Thr Leu Asn Asn Gly Ser Gln Ala Val Gly Arg Ser 
                245                 250                 255     


Ser Phe Tyr Cys Leu Glu Tyr Phe Pro Ser Gln Met Leu Arg Thr Gly 
            260                 265                 270         


Asn Asn Phe Glu Phe Ser Tyr Asn Phe Glu Asp Val Pro Phe His Ser 
        275                 280                 285             


Ser Tyr Ala His Ser Gln Ser Leu Asp Arg Leu Met Asn Pro Leu Ile 
    290                 295                 300                 


Asp Gln Tyr Leu Tyr Tyr Leu Ser Arg Thr Gln Ser Thr Gly Gly Thr 
305                 310                 315                 320 


Ala Gly Thr Gln Gln Leu Leu Phe Ser Gln Ala Gly Pro Asn Asn Met 
                325                 330                 335     


Ser Ala Gln Ala Lys Asn Trp Leu Pro Gly Pro Cys Tyr Arg Gln Gln 
            340                 345                 350         


Arg Val Ser Thr Thr Leu Ser Gln Asn Asn Asn Ser Asn Phe Ala Trp 
        355                 360                 365             


Thr Gly Ala Thr Lys Tyr His Leu Asn Gly Arg Asp Ser Leu Val Asn 
    370                 375                 380                 


Pro Gly Val Ala Met Ala Thr His Lys Asp Asp Glu Glu Arg Phe Phe 
385                 390                 395                 400 


Pro Ser Ser Gly Val Leu Met Phe Gly Lys Gln Gly Ala Gly Lys Asp 
                405                 410                 415     


Asn Val Asp Tyr Ser Ser Val Met Leu Thr Ser Glu Glu Glu Ile Lys 
            420                 425                 430         


Thr Thr Asn Pro Val Ala Thr Glu Gln Tyr Gly Val Val Ala Asp Asn 
        435                 440                 445             


Leu Gln Gln Gln Asn Ala Ala Pro Ile Val Gly Ala Val Asn Ser Gln 
    450                 455                 460                 


Gly Ala Leu Pro Gly Met Val Trp Gln Asn Arg Asp Val Tyr Leu Gln 
465                 470                 475                 480 


Gly Pro Ile Trp Ala Lys Ile Pro His Thr Asp Gly Asn Phe His Pro 
                485                 490                 495     


Ser Pro Leu Met Gly Gly Phe Gly Leu Lys His Pro Pro Pro Gln Ile 
            500                 505                 510         


Leu Ile Lys Asn Thr Pro Val Pro Ala Asp Pro Pro Thr Thr Phe Asn 
        515                 520                 525             


Gln Ala Lys Leu Ala Ser Phe Ile Thr Gln Tyr Ser Thr Gly Gln Val 
    530                 535                 540                 


Ser Val Glu Ile Glu Trp Glu Leu Gln Lys Glu Asn Ser Lys Arg Trp 
545                 550                 555                 560 


Asn Pro Glu Ile Gln Tyr Thr Ser Asn Tyr Tyr Lys Ser Thr Asn Val 
                565                 570                 575     


Asp Phe Ala Val Asn Thr Glu Gly Thr Tyr Ser Glu Pro Arg Pro Ile 
            580                 585                 590         


Gly Thr Arg Tyr Leu Thr Arg Asn Leu 
        595                 600     


<210>  4
<211>  535
<212>  PRT
<213>  Adeno-associated virus

<400>  4

Met Ala Ala Gly Gly Gly Ala Pro Met Ala Asp Asn Asn Glu Gly Ala 
1               5                   10                  15      


Asp Gly Val Gly Ser Ser Ser Gly Asn Trp His Cys Asp Ser Thr Trp 
            20                  25                  30          


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


Thr Tyr Asn Asn His Leu Tyr Lys Gln Ile Ser Asn Gly Thr Ser Gly 
    50                  55                  60                  


Gly Ser Thr Asn Asp Asn Thr Tyr Phe Gly Tyr Ser Thr Pro Trp Gly 
65                  70                  75                  80  


Tyr Phe Asp Phe Asn Arg Phe His Cys His Phe Ser Pro Arg Asp Trp 
                85                  90                  95      


Gln Arg Leu Ile Asn Asn Asn Trp Gly Phe Arg Pro Lys Arg Leu Asn 
            100                 105                 110         


Phe Lys Leu Phe Asn Ile Gln Val Lys Glu Val Thr Gln Asn Glu Gly 
        115                 120                 125             


Thr Lys Thr Ile Ala Asn Asn Leu Thr Ser Thr Ile Gln Val Phe Thr 
    130                 135                 140                 


Asp Ser Glu Tyr Gln Leu Pro Tyr Val Leu Gly Ser Ala His Gln Gly 
145                 150                 155                 160 


Cys Leu Pro Pro Phe Pro Ala Asp Val Phe Met Ile Pro Gln Tyr Gly 
                165                 170                 175     


Tyr Leu Thr Leu Asn Asn Gly Ser Gln Ala Val Gly Arg Ser Ser Phe 
            180                 185                 190         


Tyr Cys Leu Glu Tyr Phe Pro Ser Gln Met Leu Arg Thr Gly Asn Asn 
        195                 200                 205             


Phe Glu Phe Ser Tyr Asn Phe Glu Asp Val Pro Phe His Ser Ser Tyr 
    210                 215                 220                 


Ala His Ser Gln Ser Leu Asp Arg Leu Met Asn Pro Leu Ile Asp Gln 
225                 230                 235                 240 


Tyr Leu Tyr Tyr Leu Ser Arg Thr Gln Ser Thr Gly Gly Thr Ala Gly 
                245                 250                 255     


Thr Gln Gln Leu Leu Phe Ser Gln Ala Gly Pro Asn Asn Met Ser Ala 
            260                 265                 270         


Gln Ala Lys Asn Trp Leu Pro Gly Pro Cys Tyr Arg Gln Gln Arg Val 
        275                 280                 285             


Ser Thr Thr Leu Ser Gln Asn Asn Asn Ser Asn Phe Ala Trp Thr Gly 
    290                 295                 300                 


Ala Thr Lys Tyr His Leu Asn Gly Arg Asp Ser Leu Val Asn Pro Gly 
305                 310                 315                 320 


Val Ala Met Ala Thr His Lys Asp Asp Glu Glu Arg Phe Phe Pro Ser 
                325                 330                 335     


Ser Gly Val Leu Met Phe Gly Lys Gln Gly Ala Gly Lys Asp Asn Val 
            340                 345                 350         


Asp Tyr Ser Ser Val Met Leu Thr Ser Glu Glu Glu Ile Lys Thr Thr 
        355                 360                 365             


Asn Pro Val Ala Thr Glu Gln Tyr Gly Val Val Ala Asp Asn Leu Gln 
    370                 375                 380                 


Gln Gln Asn Ala Ala Pro Ile Val Gly Ala Val Asn Ser Gln Gly Ala 
385                 390                 395                 400 


Leu Pro Gly Met Val Trp Gln Asn Arg Asp Val Tyr Leu Gln Gly Pro 
                405                 410                 415     


Ile Trp Ala Lys Ile Pro His Thr Asp Gly Asn Phe His Pro Ser Pro 
            420                 425                 430         


Leu Met Gly Gly Phe Gly Leu Lys His Pro Pro Pro Gln Ile Leu Ile 
        435                 440                 445             


Lys Asn Thr Pro Val Pro Ala Asp Pro Pro Thr Thr Phe Asn Gln Ala 
    450                 455                 460                 


Lys Leu Ala Ser Phe Ile Thr Gln Tyr Ser Thr Gly Gln Val Ser Val 
465                 470                 475                 480 


Glu Ile Glu Trp Glu Leu Gln Lys Glu Asn Ser Lys Arg Trp Asn Pro 
                485                 490                 495     


Glu Ile Gln Tyr Thr Ser Asn Tyr Tyr Lys Ser Thr Asn Val Asp Phe 
            500                 505                 510         


Ala Val Asn Thr Glu Gly Thr Tyr Ser Glu Pro Arg Pro Ile Gly Thr 
        515                 520                 525             


Arg Tyr Leu Thr Arg Asn Leu 
    530                 535 


