                               SEQUENCE LISTING

<110> RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
 
<120> GENE THERAPY FOR LIMB-GIRDLE MUSCULAR DYSTROPHY TYPE 2C

<130> 106887-7141

<140>
<141>

<150> 62/624,616
<151> 2018-01-31

<160> 11    

<170> PatentIn version 3.5

<210> 1
<211> 876
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 1
atggtgaggg agcagtacac cacagcaacc gagggaatct gcatcgagag gccagagaac       60

cagtacgtgt ataagatcgg catctacggc tggcggaaga gatgtctgta tctgttcgtg      120

ctgctgctgc tgatcatcct ggtggtgaat ctggccctga ccatctggat cctgaaagtg      180

atgtggtttt ccccagcagg aatgggacac ctgtgcgtga caaaggacgg actgcggctg      240

gagggagagt ctgagttcct gtttcccctg tatgccaagg agatccacag cagagtggat      300

agctccctgc tgctgcagtc cacccagaac gtgacagtga acgcaaggaa tagcgaggga      360

gaggtgaccg gcagactgaa ggtcggcccc aagatggtgg aggtgcagaa tcagcagttc      420

cagatcaact ccaatgacgg caagcctctg tttacagtgg atgagaagga ggtggtggtg      480

ggcaccgaca agctgagggt gacaggacct gagggcgccc tgttcgagca ctctgtggag      540

accccactgg tgcgcgcaga cccttttcag gatctgaggc tggagagccc aacacgcagc      600

ctgtccatgg acgcacccag aggcgtgcac atccaggcac acgcaggcaa gatcgaggcc      660

ctgagccaga tggatatcct gttccactct agcgacggca tgctggtgct ggatgccgag      720

accgtgtgcc tgcctaagct ggtgcagggc acatggggcc catctggctc ctctcagagc      780

ctgtacgaga tctgcgtgtg cccagatggc aagctgtatc tgtccgtggc cggcgtgtct      840

accacatgcc aggagcacaa ccacatctgt ctgtga                                876


<210> 2
<211> 291
<212> PRT
<213> Homo sapiens

<400> 2
Met Val Arg Glu Gln Tyr Thr Thr Ala Thr Glu Gly Ile Cys Ile Glu 
1               5                   10                  15      


Arg Pro Glu Asn Gln Tyr Val Tyr Lys Ile Gly Ile Tyr Gly Trp Arg 
            20                  25                  30          


Lys Arg Cys Leu Tyr Leu Phe Val Leu Leu Leu Leu Ile Ile Leu Val 
        35                  40                  45              


Val Asn Leu Ala Leu Thr Ile Trp Ile Leu Lys Val Met Trp Phe Ser 
    50                  55                  60                  


Pro Ala Gly Met Gly His Leu Cys Val Thr Lys Asp Gly Leu Arg Leu 
65                  70                  75                  80  


Glu Gly Glu Ser Glu Phe Leu Phe Pro Leu Tyr Ala Lys Glu Ile His 
                85                  90                  95      


Ser Arg Val Asp Ser Ser Leu Leu Leu Gln Ser Thr Gln Asn Val Thr 
            100                 105                 110         


Val Asn Ala Arg Asn Ser Glu Gly Glu Val Thr Gly Arg Leu Lys Val 
        115                 120                 125             


Gly Pro Lys Met Val Glu Val Gln Asn Gln Gln Phe Gln Ile Asn Ser 
    130                 135                 140                 


Asn Asp Gly Lys Pro Leu Phe Thr Val Asp Glu Lys Glu Val Val Val 
145                 150                 155                 160 


Gly Thr Asp Lys Leu Arg Val Thr Gly Pro Glu Gly Ala Leu Phe Glu 
                165                 170                 175     


His Ser Val Glu Thr Pro Leu Val Arg Ala Asp Pro Phe Gln Asp Leu 
            180                 185                 190         


Arg Leu Glu Ser Pro Thr Arg Ser Leu Ser Met Asp Ala Pro Arg Gly 
        195                 200                 205             


Val His Ile Gln Ala His Ala Gly Lys Ile Glu Ala Leu Ser Gln Met 
    210                 215                 220                 


Asp Ile Leu Phe His Ser Ser Asp Gly Met Leu Val Leu Asp Ala Glu 
225                 230                 235                 240 


Thr Val Cys Leu Pro Lys Leu Val Gln Gly Thr Trp Gly Pro Ser Gly 
                245                 250                 255     


Ser Ser Gln Ser Leu Tyr Glu Ile Cys Val Cys Pro Asp Gly Lys Leu 
            260                 265                 270         


Tyr Leu Ser Val Ala Gly Val Ser Thr Thr Cys Gln Glu His Asn His 
        275                 280                 285             


Ile Cys Leu 
    290     


<210> 3
<211> 2225
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 3
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggggtt aaccaattgg      120

cgcggccgca agcttgcatg tctaagctag acccttcaga ttaaaaataa ctgaggtaag      180

ggcctgggta ggggaggtgg tgtgagacgc tcctgtctct cctctatctg cccatcggcc      240

ctttggggag gaggaatgtg cccaaggact aaaaaaaggc catggagcca gaggggcgag      300

ggcaacagac ctttcatggg caaaccttgg ggccctgctg tctagcatgc cccactacgg      360

gtctaggctg cccatgtaag gaggcaaggc ctggggacac ccgagatgcc tggttataat      420

taacccagac atgtggctgc cccccccccc ccaacacctg ctgcctctaa aaataaccct      480

gtccctggtg gatcccctgc atgcgaagat cttcgaacaa ggctgtgggg gactgagggc      540

aggctgtaac aggcttgggg gccagggctt atacgtgcct gggactccca aagtattact      600

gttccatgtt cccggcgaag ggccagctgt cccccgccag ctagactcag cacttagttt      660

aggaaccagt gagcaagtca gcccttgggg cagcccatac aaggccatgg ggctgggcaa      720

gctgcacgcc tgggtccggg gtgggcacgg tgcccgggca acgagctgaa agctcatctg      780

ctctcagggg cccctccctg gggacagccc ctcctggcta gtcacaccct gtaggctcct      840

ctatataacc caggggcaca ggggctgccc tcattctacc accacctcca cagcacagac      900

agacactcag gagcagccag cggcgcgccc aggtaagttt agtctttttg tcttttattt      960

caggtcccgg atccggtggt ggtgcaaatc aaagaactgc tcctcagtgg atgttgcctt     1020

tacttctagg cctgtacgga agtgttactt ctgctctaaa agctgcggaa ttgtacccgg     1080

taccaccatg gtgagggagc agtacaccac agcaaccgag ggaatctgca tcgagaggcc     1140

agagaaccag tacgtgtata agatcggcat ctacggctgg cggaagagat gtctgtatct     1200

gttcgtgctg ctgctgctga tcatcctggt ggtgaatctg gccctgacca tctggatcct     1260

gaaagtgatg tggttttccc cagcaggaat gggacacctg tgcgtgacaa aggacggact     1320

gcggctggag ggagagtctg agttcctgtt tcccctgtat gccaaggaga tccacagcag     1380

agtggatagc tccctgctgc tgcagtccac ccagaacgtg acagtgaacg caaggaatag     1440

cgagggagag gtgaccggca gactgaaggt cggccccaag atggtggagg tgcagaatca     1500

gcagttccag atcaactcca atgacggcaa gcctctgttt acagtggatg agaaggaggt     1560

ggtggtgggc accgacaagc tgagggtgac aggacctgag ggcgccctgt tcgagcactc     1620

tgtggagacc ccactggtgc gcgcagaccc ttttcaggat ctgaggctgg agagcccaac     1680

acgcagcctg tccatggacg cacccagagg cgtgcacatc caggcacacg caggcaagat     1740

cgaggccctg agccagatgg atatcctgtt ccactctagc gacggcatgc tggtgctgga     1800

tgccgagacc gtgtgcctgc ctaagctggt gcagggcaca tggggcccat ctggctcctc     1860

tcagagcctg tacgagatct gcgtgtgccc agatggcaag ctgtatctgt ccgtggccgg     1920

cgtgtctacc acatgccagg agcacaacca catctgtctg tgactcgagg gccgcaataa     1980

aagatcttta ttttcattag atctgtgtgt tggttttttg tgtgtcctgc aggggcgcgc     2040

ctaatctaga gcatggctac gtagataagt agcatggcgg gttaatcatt aactacaagg     2100

aacccctagt gatggagttg gccactccct ctctgcgcgc tcgctcgctc actgaggccg     2160

ggcgaccaaa ggtcgcccga cgcccgggct ttgcccgggc ggcctcagtg agcgagcgag     2220

cgcgc                                                                 2225


<210> 4
<211> 792
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 4
aagcttgcat gtctaagcta gacccttcag attaaaaata actgaggtaa gggcctgggt       60

aggggaggtg gtgtgagacg ctcctgtctc tcctctatct gcccatcggc cctttgggga      120

ggaggaatgt gcccaaggac taaaaaaagg ccatggagcc agaggggcga gggcaacaga      180

cctttcatgg gcaaaccttg gggccctgct gtctagcatg ccccactacg ggtctaggct      240

gcccatgtaa ggaggcaagg cctggggaca cccgagatgc ctggttataa ttaacccaga      300

catgtggctg cccccccccc cccaacacct gctgcctcta aaaataaccc tgtccctggt      360

ggatcccctg catgcgaaga tcttcgaaca aggctgtggg ggactgaggg caggctgtaa      420

caggcttggg ggccagggct tatacgtgcc tgggactccc aaagtattac tgttccatgt      480

tcccggcgaa gggccagctg tcccccgcca gctagactca gcacttagtt taggaaccag      540

tgagcaagtc agcccttggg gcagcccata caaggccatg gggctgggca agctgcacgc      600

ctgggtccgg ggtgggcacg gtgcccgggc aacgagctga aagctcatct gctctcaggg      660

gcccctccct ggggacagcc cctcctggct agtcacaccc tgtaggctcc tctatataac      720

ccaggggcac aggggctgcc ctcattctac caccacctcc acagcacaga cagacactca      780

ggagcagcca gc                                                          792


<210> 5
<211> 148
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 5
aggtaagttt agtctttttg tcttttattt caggtcccgg atccggtggt ggtgcaaatc       60

aaagaactgc tcctcagtgg atgttgcctt tacttctagg cctgtacgga agtgttactt      120

ctgctctaaa agctgcggaa ttgtaccc                                         148


<210> 6
<211> 53
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      oligonucleotide

<400> 6
ggccgcaata aaagatcttt attttcatta gatctgtgtg ttggtttttt gtg              53


<210> 7
<211> 110
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 7
ctgcgcgctc gctcgctcac tgaggccgcc cgggcaaagc ccgggcgtcg ggcgaccttt       60

ggtcgcccgg cctcagtgag cgagcgagcg cgcagagagg gagtggggtt                 110


<210> 8
<211> 104
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 8
ccactccctc tctgcgcgct cgctcgctca ctgaggccgg gcgaccaaag gtcgcccgac       60

gcccgggctt tgcccgggcg gcctcagtga gcgagcgagc gcgc                       104


<210> 9
<211> 876
<212> DNA
<213> Homo sapiens

<400> 9
atggtgcgtg agcagtacac tacagccaca gaaggcatct gcatagagag gccagagaat       60

cagtatgtct acaaaattgg catttatggc tggagaaagc gctgtctcta cttgtttgtt      120

cttcttttac tcatcatcct cgttgtgaat ttagctctta caatttggat tcttaaagtg      180

atgtggtttt ctccagcagg aatgggccac ttgtgtgtaa caaaagatgg actgcgcttg      240

gaaggggaat cagaattttt attcccattg tatgccaaag aaatacactc cagagtggac      300

tcatctctgc tgctacaatc aacccagaat gtgactgtaa atgcgcgcaa ctcagaaggg      360

gaggtcacag gcaggttaaa agtcggtccc aaaatggtag aagtccagaa tcaacagttt      420

cagatcaact ccaacgacgg caagccacta tttactgtag atgagaagga agttgtggtt      480

ggtacagata aacttcgagt aactgggcct gaaggggctc tttttgaaca ttcagtggag      540

acaccccttg tcagagccga cccgtttcaa gaccttagat tagaatcccc cactcggagt      600

ctaagcatgg atgccccaag gggtgtgcat attcaagctc acgctgggaa aattgaggcg      660

ctttctcaaa tggatattct ttttcatagt agtgatggaa tgcttgtgct tgatgctgaa      720

actgtgtgct tacccaagct ggtgcagggg acgtggggtc cctctggcag ctcacagagc      780

ctctacgaaa tctgtgtgtg tccagatggg aagctgtacc tgtctgtggc cggtgtgagc      840

accacgtgcc aggagcacag ccacatctgc ctctga                                876


<210> 10
<211> 535
<212> PRT
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polypeptide

<400> 10
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 


<210> 11
<211> 2217
<212> DNA
<213> Artificial Sequence

<220>
<223> Description of Artificial Sequence: Synthetic
      polynucleotide

<400> 11
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


