                         SEQUENCE LISTING

<110>  The Trustees of the University of Pennsylvania
 
<120>  COMPOSITIONS USEFUL IN TREATMENT OF SPINAL MUSCULAR ATROPY

<130>  UPN-16-7655PCT

<150>  62/267,012
<151>  2015-12-14

<160>  5     

<170>  PatentIn version 3.5

<210>  1
<211>  294
<212>  PRT
<213>  Homo sapiens

<400>  1

Met Ala Met Ser Ser Gly Gly Ser Gly Gly Gly Val Pro Glu Gln Glu 
1               5                   10                  15      


Asp Ser Val Leu Phe Arg Arg Gly Thr Gly Gln Ser Asp Asp Ser Asp 
            20                  25                  30          


Ile Trp Asp Asp Thr Ala Leu Ile Lys Ala Tyr Asp Lys Ala Val Ala 
        35                  40                  45              


Ser Phe Lys His Ala Leu Lys Asn Gly Asp Ile Cys Glu Thr Ser Gly 
    50                  55                  60                  


Lys Pro Lys Thr Thr Pro Lys Arg Lys Pro Ala Lys Lys Asn Lys Ser 
65                  70                  75                  80  


Gln Lys Lys Asn Thr Ala Ala Ser Leu Gln Gln Trp Lys Val Gly Asp 
                85                  90                  95      


Lys Cys Ser Ala Ile Trp Ser Glu Asp Gly Cys Ile Tyr Pro Ala Thr 
            100                 105                 110         


Ile Ala Ser Ile Asp Phe Lys Arg Glu Thr Cys Val Val Val Tyr Thr 
        115                 120                 125             


Gly Tyr Gly Asn Arg Glu Glu Gln Asn Leu Ser Asp Leu Leu Ser Pro 
    130                 135                 140                 


Ile Cys Glu Val Ala Asn Asn Ile Glu Gln Asn Ala Gln Glu Asn Glu 
145                 150                 155                 160 


Asn Glu Ser Gln Val Ser Thr Asp Glu Ser Glu Asn Ser Arg Ser Pro 
                165                 170                 175     


Gly Asn Lys Ser Asp Asn Ile Lys Pro Lys Ser Ala Pro Trp Asn Ser 
            180                 185                 190         


Phe Leu Pro Pro Pro Pro Pro Met Pro Gly Pro Arg Leu Gly Pro Gly 
        195                 200                 205             


Lys Pro Gly Leu Lys Phe Asn Gly Pro Pro Pro Pro Pro Pro Pro Pro 
    210                 215                 220                 


Pro Pro His Leu Leu Ser Cys Trp Leu Pro Pro Phe Pro Ser Gly Pro 
225                 230                 235                 240 


Pro Ile Ile Pro Pro Pro Pro Pro Ile Cys Pro Asp Ser Leu Asp Asp 
                245                 250                 255     


Ala Asp Ala Leu Gly Ser Met Leu Ile Ser Trp Tyr Met Ser Gly Tyr 
            260                 265                 270         


His Thr Gly Tyr Tyr Met Gly Phe Arg Gln Asn Gln Lys Glu Gly Arg 
        275                 280                 285             


Cys Ser His Ser Leu Asn 
    290                 


<210>  2
<211>  882
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  constructed sequence

<400>  2
atggccatgt cgagtggggg cagtggaggg ggagtgccag aacaggaaga ttccgtgctg       60

ttcaggcgag gaaccgggca gagtgacgac agtgacattt gggacgacac ggccctgatc      120

aaggcctatg acaaagccgt ggcctccttc aagcacgcgc tgaagaacgg cgacatttgc      180

gaaaccagcg gcaagcctaa gaccacccct aaacggaagc ccgccaagaa aaataagtcc      240

cagaaaaaga acacagccgc aagtcttcag caatggaagg tgggggataa gtgctccgcg      300

atatggagtg aagacgggtg catctatcct gccaccatcg ccagcataga cttcaagcgc      360

gaaacctgcg tggtggtgta cactggatac gggaaccggg aggagcagaa cctgagcgac      420

ctgttgagcc ctatttgtga ggtggccaac aacatcgagc agaatgcgca agaaaatgaa      480

aacgagagtc aggtgtccac cgatgagagt gaaaacagta ggagccccgg caacaaatcc      540

gacaatatca agcccaaaag cgcaccctgg aatagcttcc ttccaccccc ccccccaatg      600

cccggacctc gactgggccc cggaaagcct ggcctgaagt tcaacggccc ccctcctcct      660

cctccccctc ctccccccca cctgctgagc tgctggttgc cccctttccc ttcgggaccc      720

cctatcatac ctcccccccc ccctatttgc cctgactccc tggacgacgc ggacgcgctg      780

ggcagtatgc tcatctcgtg gtacatgtca ggataccaca ccgggtacta catgggcttc      840

agacaaaatc agaaggaagg acgatgtagt cactccctga at                         882


<210>  3
<211>  885
<212>  DNA
<213>  Homo sapiens

<400>  3
atggcgatga gcagcggcgg cagtggtggc ggcgtcccgg agcaggagga ttccgtgctg       60

ttccggcgcg gcacaggcca gagcgatgat tctgacattt gggatgatac agcactgata      120

aaagcatatg ataaagctgt ggcttcattt aagcatgctc taaagaatgg tgacatttgt      180

gaaacttcgg gtaaaccaaa aaccacacct aaaagaaaac ctgctaagaa gaataaaagc      240

caaaagaaga atactgcagc ttccttacaa cagtggaaag ttggggacaa atgttctgcc      300

atttggtcag aagacggttg catttaccca gctaccattg cttcaattga ttttaagaga      360

gaaacctgtg ttgtggttta cactggatat ggaaatagag aggagcaaaa tctgtccgat      420

ctactttccc caatctgtga agtagctaat aatatagaac aaaatgctca agagaatgaa      480

aatgaaagcc aagtttcaac agatgaaagt gagaactcca ggtctcctgg aaataaatca      540

gataacatca agcccaaatc tgctccatgg aactcttttc tccctccacc accccccatg      600

ccagggccaa gactgggacc aggaaagcca ggtctaaaat tcaatggccc accaccgcca      660

ccgccaccac caccacccca cttactatca tgctggctgc ctccatttcc ttctggacca      720

ccaataattc ccccaccacc tcccatatgt ccagattctc ttgatgatgc tgatgctttg      780

ggaagtatgt taatttcatg gtacatgagt ggctatcata ctggctatta tatgggtttc      840

agacaaaatc aaaaagaagg aaggtgctca cattccttaa attaa                      885


<210>  4
<211>  6079
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  constructed sequence

<400>  4
aattctagct tgccaccatg gccatgtcga gtgggggcag tggaggggga gtgccagaac       60

aggaagattc cgtgctgttc aggcgaggaa ccgggcagag tgacgacagt gacatttggg      120

acgacacggc cctgatcaag gcctatgaca aagccgtggc ctccttcaag cacgcgctga      180

agaacggcga catttgcgaa accagcggca agcctaagac cacccctaaa cggaagcccg      240

ccaagaaaaa taagtcccag aaaaagaaca cagccgcaag tcttcagcaa tggaaggtgg      300

gggataagtg ctccgcgata tggagtgaag acgggtgcat ctatcctgcc accatcgcca      360

gcatagactt caagcgcgaa acctgcgtgg tggtgtacac tggatacggg aaccgggagg      420

agcagaacct gagcgacctg ttgagcccta tttgtgaggt ggccaacaac atcgagcaga      480

atgcgcaaga aaatgaaaac gagagtcagg tgtccaccga tgagagtgaa aacagtagga      540

gccccggcaa caaatccgac aatatcaagc ccaaaagcgc accctggaat agcttccttc      600

cacccccccc cccaatgccc ggacctcgac tgggccccgg aaagcctggc ctgaagttca      660

acggcccccc tcctcctcct ccccctcctc ccccccacct gctgagctgc tggttgcccc      720

ctttcccttc gggaccccct atcatacctc cccccccccc tatttgccct gactccctgg      780

acgacgcgga cgcgctgggc agtatgctca tctcgtggta catgtcagga taccacaccg      840

ggtactacat gggcttcaga caaaatcaga aggaaggacg atgtagtcac tccctgaatt      900

aatgatagct agaattcacg cgtggtacct ctagagtcga cccgggcggc ctcgaggacg      960

gggtgaacta cgcctgagga tccgatcttt ttccctctgc caaaaattat ggggacatca     1020

tgaagcccct tgagcatctg acttctggct aataaaggaa atttattttc attgcaatag     1080

tgtgttggaa ttttttgtgt ctctcactcg gaagcaattc gttgatctga atttcgacca     1140

cccataatac ccattaccct ggtagataag tagcatggcg ggttaatcat taactacaag     1200

gaacccctag tgatggagtt ggccactccc tctctgcgcg ctcgctcgct cactgaggcc     1260

gggcgaccaa aggtcgcccg acgcccgggc tttgcccggg cggcctcagt gagcgagcga     1320

gcgcgcagcc ttaattaacc taattcactg gccgtcgttt tacaacgtcg tgactgggaa     1380

aaccctggcg ttacccaact taatcgcctt gcagcacatc cccctttcgc cagctggcgt     1440

aatagcgaag aggcccgcac cgatcgccct tcccaacagt tgcgcagcct gaatggcgaa     1500

tgggacgcgc cctgtagcgg cgcattaagc gcggcgggtg tggtggttac gcgcagcgtg     1560

accgctacac ttgccagcgc cctagcgccc gctcctttcg ctttcttccc ttcctttctc     1620

gccacgttcg ccggctttcc ccgtcaagct ctaaatcggg ggctcccttt agggttccga     1680

tttagtgctt tacggcacct cgaccccaaa aaacttgatt agggtgatgg ttcacgtagt     1740

gggccatcgc cctgatagac ggtttttcgc cctttgacgt tggagtccac gttctttaat     1800

agtggactct tgttccaaac tggaacaaca ctcaacccta tctcggtcta ttcttttgat     1860

ttataaggga ttttgccgat ttcggcctat tggttaaaaa atgagctgat ttaacaaaaa     1920

tttaacgcga attttaacaa aatattaacg cttacaattt aggtggcact tttcggggaa     1980

atgtgcgcgg aacccctatt tgtttatttt tctaaataca ttcaaatatg tatccgctca     2040

tgagacaata accctgataa atgcttcaat aatattgaaa aaggaagagt atgagtattc     2100

aacatttccg tgtcgccctt attccctttt ttgcggcatt ttgccttcct gtttttgctc     2160

acccagaaac gctggtgaaa gtaaaagatg ctgaagatca gttgggtgca cgagtgggtt     2220

acatcgaact ggatctcaac agcggtaaga tccttgagag ttttcgcccc gaagaacgtt     2280

ttccaatgat gagcactttt aaagttctgc tatgtggcgc ggtattatcc cgtattgacg     2340

ccgggcaaga gcaactcggt cgccgcatac actattctca gaatgacttg gttgagtact     2400

caccagtcac agaaaagcat cttacggatg gcatgacagt aagagaatta tgcagtgctg     2460

ccataaccat gagtgataac actgcggcca acttacttct gacaacgatc ggaggaccga     2520

aggagctaac cgcttttttg cacaacatgg gggatcatgt aactcgcctt gatcgttggg     2580

aaccggagct gaatgaagcc ataccaaacg acgagcgtga caccacgatg cctgtagcaa     2640

tggcaacaac gttgcgcaaa ctattaactg gcgaactact tactctagct tcccggcaac     2700

aattaataga ctggatggag gcggataaag ttgcaggacc acttctgcgc tcggcccttc     2760

cggctggctg gtttattgct gataaatctg gagccggtga gcgtgggtct cgcggtatca     2820

ttgcagcact ggggccagat ggtaagccct cccgtatcgt agttatctac acgacgggga     2880

gtcaggcaac tatggatgaa cgaaatagac agatcgctga gataggtgcc tcactgatta     2940

agcattggta actgtcagac caagtttact catatatact ttagattgat ttaaaacttc     3000

atttttaatt taaaaggatc taggtgaaga tcctttttga taatctcatg accaaaatcc     3060

cttaacgtga gttttcgttc cactgagcgt cagaccccgt agaaaagatc aaaggatctt     3120

cttgagatcc tttttttctg cgcgtaatct gctgcttgca aacaaaaaaa ccaccgctac     3180

cagcggtggt ttgtttgccg gatcaagagc taccaactct ttttccgaag gtaactggct     3240

tcagcagagc gcagatacca aatactgttc ttctagtgta gccgtagtta ggccaccact     3300

tcaagaactc tgtagcaccg cctacatacc tcgctctgct aatcctgtta ccagtggctg     3360

ctgccagtgg cgataagtcg tgtcttaccg ggttggactc aagacgatag ttaccggata     3420

aggcgcagcg gtcgggctga acggggggtt cgtgcacaca gcccagcttg gagcgaacga     3480

cctacaccga actgagatac ctacagcgtg agctatgaga aagcgccacg cttcccgaag     3540

ggagaaaggc ggacaggtat ccggtaagcg gcagggtcgg aacaggagag cgcacgaggg     3600

agcttccagg gggaaacgcc tggtatcttt atagtcctgt cgggtttcgc cacctctgac     3660

ttgagcgtcg atttttgtga tgctcgtcag gggggcggag cctatggaaa aacgccagca     3720

acgcggcctt tttacggttc ctggcctttt gctggccttt tgctcacatg ttctttcctg     3780

cgttatcccc tgattctgtg gataaccgta ttaccgcctt tgagtgagct gataccgctc     3840

gccgcagccg aacgaccgag cgcagcgagt cagtgagcga ggaagcggaa gagcgcccaa     3900

tacgcaaacc gcctctcccc gcgcgttggc cgattcatta atgcagctgg cacgacaggt     3960

ttcccgactg gaaagcgggc agtgagcgca acgcaattaa tgtgagttag ctcactcatt     4020

aggcacccca ggctttacac tttatgcttc cggctcgtat gttgtgtgga attgtgagcg     4080

gataacaatt tcacacagga aacagctatg accatgatta cgccagattt aattaaggcc     4140

ttaattaggc tgcgcgctcg ctcgctcact gaggccgccc gggcaaagcc cgggcgtcgg     4200

gcgacctttg gtcgcccggc ctcagtgagc gagcgagcgc gcagagaggg agtggccaac     4260

tccatcacta ggggttcctt gtagttaatg attaacccgc catgctactt atctaccagg     4320

gtaatgggga tcctctagaa ctatagctag tcgacattga ttattgacta gttattaata     4380

gtaatcaatt acggggtcat tagttcatag cccatatatg gagttccgcg ttacataact     4440

tacggtaaat ggcccgcctg gctgaccgcc caacgacccc cgcccattga cgtcaataat     4500

gacgtatgtt cccatagtaa cgccaatagg gactttccat tgacgtcaat gggtggacta     4560

tttacggtaa actgcccact tggcagtaca tcaagtgtat catatgccaa gtacgccccc     4620

tattgacgtc aatgacggta aatggcccgc ctggcattat gcccagtaca tgaccttatg     4680

ggactttcct acttggcagt acatctacgt attagtcatc gctattacca tggtcgaggt     4740

gagccccacg ttctgcttca ctctccccat ctcccccccc tccccacccc caattttgta     4800

tttatttatt ttttaattat tttgtgcagc gatgggggcg gggggggggg gggggcgcgc     4860

gccaggcggg gcggggcggg gcgaggggcg gggcggggcg aggcggagag gtgcggcggc     4920

agccaatcag agcggcgcgc tccgaaagtt tccttttatg gcgaggcggc ggcggcggcg     4980

gccctataaa aagcgaagcg cgcggcgggc ggggagtcgc tgcgacgctg ccttcgcccc     5040

gtgccccgct ccgccgccgc ctcgcgccgc ccgccccggc tctgactgac cgcgttactc     5100

ccacaggtga gcgggcggga cggcccttct cctccgggct gtaattagcg cttggtttaa     5160

tgacggcttg tttcttttct gtggctgcgt gaaagccttg aggggctccg ggagggccct     5220

ttgtgcgggg ggagcggctc ggggggtgcg tgcgtgtgtg tgtgcgtggg gagcgccgcg     5280

tgcggctccg cgctgcccgg cggctgtgag cgctgcgggc gcggcgcggg gctttgtgcg     5340

ctccgcagtg tgcgcgaggg gagcgcggcc gggggcggtg ccccgcggtg cggggggggc     5400

tgcgagggga acaaaggctg cgtgcggggt gtgtgcgtgg gggggtgagc agggggtgtg     5460

ggcgcgtcgg tcgggctgca accccccctg cacccccctc cccgagttgc tgagcacggc     5520

ccggcttcgg gtgcggggct ccgtacgggg cgtggcgcgg ggctcgccgt gccgggcggg     5580

gggtggcggc aggtgggggt gccgggcggg gcggggccgc ctcgggccgg ggagggctcg     5640

ggggaggggc gcggcggccc ccggagcgcc ggcggctgtc gaggcgcggc gagccgcagc     5700

cattgccttt tatggtaatc gtgcgagagg gcgcagggac ttcctttgtc ccaaatctgt     5760

gcggagccga aatctgggag gcgccgccgc accccctcta gcgggcgcgg ggcgaagcgg     5820

tgcggcgccg gcaggaagga aatgggcggg gagggccttc gtgcgtcgcc gcgccgccgt     5880

ccccttctcc ctctccagcc tcggggctgt ccgcgggggg acggctgcct tcggggggga     5940

cggggcaggg cggggttcgg cttctggcgt gtgaccggcg gctctagagc ctctgctaac     6000

catgttcatg ccttcttctt tttcctacag ctcctgggca acgtgctggt tattgtgctg     6060

tctcatcatt ttggcaaag                                                  6079


<210>  5
<211>  738
<212>  PRT
<213>  Artificial sequence

<220>
<223>  Adeno-associated virus rh10 VP1 protein

<400>  5

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 Asp 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 Lys 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 Glu Gly Ala 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     


Gln 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 Ser 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 Asp 
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 
        


