
                                SEQUENCE LISTING

<110> PTC THERAPEUTICS, INC.

<120> COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY

<130> 10589-162-228

<140> PCT/US2013/025292    
<141> 2013-02-08  

<150> 61/597,523           
<151> 2012-02-10  

<160> 21

<170> FastSEQ for Windows Version 4.0

<210> 1
<211> 19
<212> DNA
<213> Artificial Sequence

<220> 
<223> SMN Forward Primer A

<400> 1
gaaggaaggt gctcacatt                                              19

<210> 2
<211> 22
<212> DNA
<213> Artificial Sequence

<220> 
<223> SMN Reverse Primer A

<400> 2
tctttatgtt tttggcgtct tc                                          22

<210> 3
<211> 26
<212> DNA
<213> Artificial Sequence

<220> 
<223> SMN Forward Probe A

<400> 3
aaggagaaat gctggcatag agcagc                                      26

<210> 4
<211> 21
<212> DNA
<213> Artificial Sequence

<220> 
<223> hGAPDH Forward Probe

<400> 4
cgcctggtca ccagggctgc t                                           21

<210> 5
<211> 21
<212> DNA
<213> Artificial Sequence

<220> 
<223> hGAPDH Forward Primer

<400> 5
caacggattt ggtcgtattg g                                           21

<210> 6
<211> 25
<212> DNA
<213> Artificial Sequence

<220> 
<223> hGAPDH Reverse Primer

<400> 6
tgatggcaac aatatccact ttacc                                       25

<210> 7
<211> 27
<212> DNA
<213> Artificial Sequence

<220> 
<223> SMN FL Forward Primer B

<400> 7
gctcacattc cttaaattaa ggagaaa                                     27

<210> 8
<211> 28
<212> DNA
<213> Artificial Sequence

<220> 
<223> SMN delta-7 Forward Primer B

<400> 8
tggctatcat actggctatt atatggaa                                    28

<210> 9
<211> 24
<212> DNA
<213> Artificial Sequence

<220> 
<223> SMN Reverse Primer B

<400> 9
tccagatctg tctgatcgtt tctt                                        24

<210> 10
<211> 30
<212> DNA
<213> Artificial Sequence

<220> 
<223> SMN Forward Probe B

<400> 10
ctggcataga gcagcactaa atgacaccac                                  30

<210> 11
<211> 21
<212> DNA
<213> Artificial Sequence

<220> 
<223> SMN Forward C

<400> 11
gatgctgatg ctttgggaag t                                           21

<210> 12
<211> 22
<212> DNA
<213> Artificial Sequence

<220> 
<223> SMN Reverse C

<400> 12
cgcttcacat tccagatctg tc                                          22

<210> 13
<211> 25
<212> DNA
<213> Artificial Sequence

<220> 
<223> SMN Forward D

<400> 13
atatgtccag attctcttga tgatg                                       25

<210> 14
<211> 11
<212> DNA
<213> Artificial Sequence

<220> 
<223> 5-prime end of exon 6 of the SMN2 gene 

<400> 14
ataattcccc c                                                      11

<210> 15
<211> 6
<212> DNA
<213> Artificial Sequence

<220> 
<223> nucleic acid residue 23 of exon 8 of the SMN2 gene 

<400> 15
cagcac                                                            6

<210> 16
<211> 28
<212> DNA
<213> Artificial Sequence

<220> 
<223> PCR forward primer

<400> 16
cgcggatcca taattccccc accacctc                                    28

<210> 17
<211> 28
<212> DNA
<213> Artificial Sequence

<220> 
<223> PCR reverse primer

<400> 17
cgcggatccg tgctgctcta tgccagca                                    28

<210> 18
<211> 6
<212> DNA
<213> Artificial Sequence

<220> 
<223> BamHI restriction endonuclease recognition sequence

<400> 18
ggatcc                                                            6

<210> 19
<211> 70
<212> DNA
<213> Artificial Sequence

<220> 
<223> 5-prime DEG UTR

<400> 19
tagcttctta cccgtactcc accgttggca gcacgatcgc acgtcccacg tgaaccattg 60
gtaaaccctg                                                        70

<210> 20
<211> 120
<212> DNA
<213> Artificial Sequence

<220> 
<223> 3-prime DEG UTR

<400> 20
atcgaaagta caggactagc cttcctagca accgcgggct gggagtctga gacatcactc 60
aagatatatg ctcggtaacg tatgctctag ccatctaact attccctatg tcttataggg 120


<210> 21
<211> 8266
<212> DNA
<213> Artificial Sequence

<220> 
<223> DNA sequence of SMN2-A minigene

<400> 21
tagcttctta cccgtactcc accgttggca gcacgatcgc acgtcccacg tgaaccattg 60
gtaaaccctg atgggatcca taattccccc accacctccc atatgtccag attctcttga 120
tgatgctgat gctttgggaa gtatgttaat ttcatggtac atgagtggct atcatactgg 180
ctattatatg gtaagtaatc actcagcatc ttttcctgac aatttttttg tagttatgtg 240
actttgtttt gtaaatttat aaaatactac ttgcttctct ctttatatta ctaaaaaata 300
aaaataaaaa aatacaactg tctgaggctt aaattactct tgcattgtcc ctaagtataa 360
ttttagttaa ttttaaaaag ctttcatgct attgttagat tattttgatt atacactttt 420
gaattgaaat tatacttttt ctaaataatg ttttaatctc tgatttgaaa ttgattgtag 480
ggaatggaaa agatgggata atttttcata aatgaaaaat gaaattcttt tttttttttt 540
tttttttttg agacggagtc ttgctctgtt gcccaggctg gagtgcaatg gcgtgatctt 600
ggctcacagc aagctctgcc tcctggattc acgccattct cctgcctcag cctcagaggt 660
agctgggact acaggtgcct gccaccacgc ctgtctaatt ttttgtattt ttttgtaaag 720
acagggtttc actgtgttag ccaggatggt ctcaatctcc tgaccccgtg atccacccgc 780
ctcggccttc caagagaaat gaaatttttt taatgcacaa agatctgggg taatgtgtac 840
cacattgaac cttggggagt atggcttcaa acttgtcact ttatacgtta gtctcctacg 900
gacatgttct attgtatttt agtcagaaca tttaaaatta ttttatttta ttttattttt 960
tttttttttt tgagacggag tctcgctctg tcacccaggc tggagtacag tggcgcagtc 1020
tcggctcact gcaagctccg cctcccgggt tcacgccatt ctcctgcctc agcctctccg 1080
agtagctggg actacaggcg cccgccacca cgcccggcta attttttttt atttttagta 1140
gagacggggt ttcaccgtgg tctcgatctc ctgacctcgt gatccacccg cctcggcctc 1200
ccaaagtgct gggattacaa gcgtgagcca ccgcgcccgg cctaaaatta tttttaaaag 1260
taagctcttg tgccctgcta aaattatgat gtgatattgt aggcacttgt atttttagta 1320
aattaatata gaagaaacaa ctgacttaaa ggtgtatgtt tttaaatgta tcatctgtgt 1380
gtgcccccat taatattctt atttaaaagt taaggccaga catggtggct tacaactgta 1440
atcccaacag tttgtgaggc cgaggcaggc agatcacttg aggtcaggag tttgagacca 1500
gcctggccaa catgatgaaa ccttgtctct actaaaaata ccaaaaaaaa tttagccagg 1560
catggtggca catgcctgta atccgagcta cttgggaggc tgtggcagga aaattgcttt 1620
aatctgggag gcagaggttg cagtgagttg agattgtgcc actgcactcc acccttggtg 1680
acagagtgag attccatctc aaaaaaagaa aaaggcctgg cacggtggct cacacctata 1740
atcccagtac tttgggaggt agaggcaggt ggatcacttg aggttaggag ttcaggacca 1800
gcctggccaa catggtgact actccatttc tactaaatac acaaaactta gcccagtggc 1860
gggcagttgt aatcccagct acttgagagg ttgaggcagg agaatcactt gaacctggga 1920
ggcagaggtt gcagtgagcc gagatcacac cgctgcactc tagcctggcc aacagagtga 1980
gaatttgcgg agggaaaaaa aagtcacgct tcagttgttg tagtataacc ttggtatatt 2040
gtatgtatca tgaattcctc attttaatga ccaaaaagta ataaatcaac agcttgtaat 2100
ttgttttgag atcagttatc tgactgtaac actgtaggct tttgtgtttt ttaaattatg 2160
aaatatttga aaaaaataca taatgtatat ataaagtatt ggtataattt atgttctaaa 2220
taactttctt gagaaataat tcacatggtg tgcagtttac ctttgaaagt atacaagttg 2280
gctgggcaca atggctcacg cctgtaatcc cagcactttg ggaggccagg gcaggtggat 2340
cacgaggtca ggagatcgag accatcctgg ctaacatggt gaaaccccgt ctctactaaa 2400
agtacaaaaa caaattagcc gggcatgttg gcgggcacct tttgtcccag ctgctcggga 2460
ggctgaggca ggagagtggc gtgaacccag gaggtggagc ttgcagtgag ccgagattgt 2520
gccagtgcac tccagcctgg gcgacagagc gagactctgt ctcaaaaaat aaaataaaaa 2580
agaaagtata caagtcagtg gttttggttt tcagttatgc aaccatcact acaatttaag 2640
aacattttca tcaccccaaa aagaaaccct gttaccttca ttttccccag ccctaggcag 2700
tcagtacact ttctgtctct atgaatttgt ctattttaga tattatatat aaacggaatt 2760
atacgatatg tggtcttttg tgtctggctt ctttcactta gcatgctatt ttcaagattc 2820
atccatgctg tagaatgcac cagtactgca ttccttctta ttgctgaata ttctgttgtt 2880
tggttatatc acattttatc cattcatcag ttcatggaca tttaggttgt ttttattttt 2940
gggctataat gaataatgtt gctatgaaca ttcgtttgtg ttctttttgt ttttttggtt 3000
ttttgggttt tttttgtttt gtttttgttt ttgagacagt cttgctctgt ctcctaagct 3060
ggagtgcagt ggcatgatct tggcttactg caagctctgc ctcccgggtt cacaccattc 3120
tcctgcctca gcccgacaag tagctgggac tacaggcgtg tgccaccatg cacggctaat 3180
tttttgtatt tttagtagag atggggtttc accgtgttag ccaggatggt ctcgatctcc 3240
tgacctcgtg atctgcctgc ctaggcctcc caaagtgctg ggattacagg cgtgagccac 3300
tgcacctggc cttaagtgtt tttaatacgt cattgcctta agctaacaat tcttaacctt 3360
tgttctactg aagccacgtg gttgagatag gctctgagtc tagcttttaa cctctatctt 3420
tttgtcttag aaatctaagc agaatgcaaa tgactaagaa taatgttgtt gaaataacat 3480
aaaataggtt ataactttga tactcattag taacaaatct ttcaatacat cttacggtct 3540
gttaggtgta gattagtaat gaagtgggaa gccactgcaa gctagtatac atgtagggaa 3600
agatagaaag cattgaagcc agaagagaga cagaggacat ttgggctaga tctgacaaga 3660
aaaacaaatg ttttagtatt aatttttgac tttaaatttt ttttttattt agtgaatact 3720
ggtgtttaat ggtctcattt taataagtat gacacaggta gtttaaggtc atatatttta 3780
tttgatgaaa ataaggtata ggccgggcac ggtggctcac acctgtaatc ccagcacttt 3840
gggaggccga ggcaggcgga tcacctgagg tcgggagtta gagactagcc tcaacatgga 3900
gaaaccccgt ctctactaaa aaaaatacaa aattaggcgg gcgtggtggt gcatgcctgt 3960
aatcccagct actcaggagg ctgaggcagg agaattgctt gaacctggga ggtggaggtt 4020
gcggtgagcc gagatcacct cattgcactc cagcctgggc aacaagagca aaactccatc 4080
tcaaaaaaaa aaaaataagg tataagcggg ctcaggaaca tcattggaca tactgaaaga 4140
agaaaaatca gctgggcgca gtggctcacg ccggtaatcc caacactttg ggaggccaag 4200
gcaggcgaat cacctgaagt cgggagttcc agatcagcct gaccaacatg gagaaaccct 4260
gtctctacta aaaatacaaa actagccggg catggtggcg catgcctgta atcccagcta 4320
cttgggaggc tgaggcagga gaattgcttg aaccgagaag gcggaggttg cggtgagcca 4380
agattgcacc attgcactcc agcctgggca acaagagcga aactccgtct caaaaaaaaa 4440
aggaagaaaa atattttttt aaattaatta gtttatttat tttttaagat ggagttttgc 4500
cctgtcaccc aggctggggt gcaatggtgc aatctcggct cactgcaacc tccgcctcct 4560
gggttcaagt gattctcctg cctcagcttc ccgagtagct gtgattacag ccatatgcca 4620
ccacgcccag ccagttttgt gttttgtttt gttttttgtt tttttttttt gagagggtgt 4680
cttgctctgt cccccaagct ggagtgcagc ggcgcgatct tggctcactg caagctctgc 4740
ctcccaggtt cacaccattc tcttgcctca gcctcccgag tagctgggac tacaggtgcc 4800
cgccaccaca cccggctaat ttttttgtgt ttttagtaga gatggggttt cactgtgtta 4860
gccaggatgg tctcgatctc ctgacctttt gatccacccg cctcagcctc cccaagtgct 4920
gggattatag gcgtgagcca ctgtgcccgg cctagtcttg tatttttagt agagtcggga 4980
tttctccatg ttggtcaggc tgttctccaa atccgacctc aggtgatccg cccgccttgg 5040
cctccaaaag tgcaaggcaa ggcattacag gcatgagcca ctgtgaccgg caatgttttt 5100
aaatttttta catttaaatt ttatttttta gagaccaggt ctcactctat tgctcaggct 5160
ggagtgcaag ggcacattca cagctcactg cagccttgac ctccagggct caagcagtcc 5220
tctcacctca gtttcccgag tagctgggac tacagtgata atgccactgc acctggctaa 5280
tttttatttt tatttattta tttttttttg agacagagtc ttgctctgtc acccaggctg 5340
gagtgcagtg gtgtaaatct cagctcactg cagcctccgc ctcctgggtt caagtgattc 5400
tcctgcctca acctcccaag tagctgggat tagaggtccc caccaccatg cctggctaat 5460
tttttgtact ttcagtagaa acggggtttt gccatgttgg ccaggctgtt ctcgaactcc 5520
tgagctcagg tgatccaact gtctcggcct cccaaagtgc tgggattaca ggcgtgagcc 5580
actgtgccta gcctgagcca ccacgccggc ctaattttta aattttttgt agagacaggg 5640
tctcattatg ttgcccaggg tggtgtcaag ctccaggtct caagtgatcc ccctacctcc 5700
gcctcccaaa gttgtgggat tgtaggcatg agccactgca agaaaacctt aactgcagcc 5760
taataattgt tttctttggg ataactttta aagtacatta aaagactatc aacttaattt 5820
ctgatcatat tttgttgaat aaaataagta aaatgtcttg tgaaacaaaa tgctttttaa 5880
catccatata aagctatcta tatatagcta tctatatcta tatagctatt ttttttaact 5940
tcctttattt tccttacagg gttttagaca aaatcaaaaa gaaggaaggt gctcacattc 6000
cttaaatata aggagtaagt ctgccagcat tatgaaagtg aatcttactt ttgtaaaact 6060
ttatggtttg tggaaaacaa atgtttttga acatttaaaa agttcagatg ttagaaagtt 6120
gaaaggttaa tgtaaaacaa tcaatattaa agaattttga tgccaaaact attagataaa 6180
aggttaatct acatccctac tagaattctc atacttaact ggttggttgt gtggaagaaa 6240
catactttca caataaagag ctttaggata tgatgccatt ttatatcact agtaggcaga 6300
ccagcagact tttttttatt gtgatatggg ataacctagg catactgcac tgtacactct 6360
gacatatgaa gtgctctagt caagtttaac tggtgtccac agaggacatg gtttaactgg 6420
aattcgtcaa gcctctggtt ctaatttctc atttgcagga aatgctggca tagagcagca 6480
cggatccgaa gacgccaaaa acataaagaa aggcccggcg ccattctatc ctctagagga 6540
tggaaccgct ggagagcaac tgcataaggc tatgaagaga tacgccctgg ttcctggaac 6600
aattgctttt acagatgcac atatcgaggt gaacatcacg tacgcggaat acttcgaaat 6660
gtccgttcgg ttggcagaag ctatgaaacg atatgggctg aatacaaatc acagaatcgt 6720
cgtatgcagt gaaaactctc ttcaattctt tatgccggtg ttgggcgcgt tatttatcgg 6780
agttgcagtt gcgcccgcga acgacattta taatgaacgt gaattgctca acagtatgaa 6840
catttcgcag cctaccgtag tgtttgtttc caaaaagggg ttgcaaaaaa ttttgaacgt 6900
gcaaaaaaaa ttaccaataa tccagaaaat tattatcatg gattctaaaa cggattacca 6960
gggatttcag tcgatgtaca cgttcgtcac atctcatcta cctcccggtt ttaatgaata 7020
cgattttgta ccagagtcct ttgatcgtga caaaacaatt gcactgataa tgaattcctc 7080
tggatctact gggttaccta agggtgtggc ccttccgcat agaactgcct gcgtcagatt 7140
ctcgcatgcc agagatccta tttttggcaa tcaaatcatt ccggatactg cgattttaag 7200
tgttgttcca ttccatcacg gttttggaat gtttactaca ctcggatatt tgatatgtgg 7260
atttcgagtc gtcttaatgt atagatttga agaagagctg tttttacgat cccttcagga 7320
ttacaaaatt caaagtgcgt tgctagtacc aaccctattt tcattcttcg ccaaaagcac 7380
tctgattgac aaatacgatt tatctaattt acacgaaatt gcttctgggg gcgcacctct 7440
ttcgaaagaa gtcggggaag cggttgcaaa acgcttccat cttccaggga tacgacaagg 7500
atatgggctc actgagacta catcagctat tctgattaca cccgaggggg atgataaacc 7560
gggcgcggtc ggtaaagttg ttccattttt tgaagcgaag gttgtggatc tggataccgg 7620
gaaaacgctg ggcgttaatc agagaggcga attatgtgtc agaggaccta tgattatgtc 7680
cggttatgta aacaatccgg aagcgaccaa cgccttgatt gacaaggatg gatggctaca 7740
ttctggagac atagcttact gggacgaaga cgaacacttc ttcatagttg accgcttgaa 7800
gtctttaatt aaatacaaag gatatcaggt ggcccccgct gaattggaat cgatattgtt 7860
acaacacccc aacatcttcg acgcgggcgt ggcaggtctt cccgacgatg acgccggtga 7920
acttcccgcc gccgttgttg ttttggagca cggaaagacg atgacggaaa aagagatcgt 7980
ggattacgtc gccagtcaag taacaaccgc gaaaaagttg cgcggaggag ttgtgtttgt 8040
ggacgaagta ccgaaaggtc ttaccggaaa actcgacgca agaaaaatca gagagatcct 8100
cataaaggcc aagaagggcg gaaagtccaa attgcgcggc cgctaaatcg aaagtacagg 8160
actagccttc ctagcaaccg cgggctggga gtctgagaca tcactcaaga tatatgctcg 8220
gtaacgtatg ctctagccat ctaactattc cctatgtctt ataggg                8266


