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Sequences

Sequence NM_138821

GTTCTGAATGATGACTGACGCGGGTTTGGGTGATACCCCTCACAGCCCCTGTCATTCCGGAGTCATAAGGCACCCGCGCGTCTAGCCCCAGCGCCAGGGC ACGCGAGCGGCGCTGGAGGGAGGAAAGCTTCCGCCTGCGGGCCGGACAAAAGTCCCGCCTGCCCACGGCTTTTTGCCCGCCGCTCGTGACCGAGACGCCT CGCCGCGGCCAGCTCGCTGCTCTCGCTGGCGGATGGTGTGTGGCCGCCGCAGGACGCCCGCCGTGCCCGGGCCATGAAGTAGCGGCTGCTGGCGGCGCCG CTGCCCAACCGCCAGCCCCAGCCCCGCGCTGCGCTGCCCGGTCCTCTCCCGGCGGGGTCGTATCGGCGTGGACATGGCTGGCCGCGTCCCTAGCCTGCTA GTTCTCCTTGTTTTTCCAAGCAGCTGTTTGGCTTTCCGAAGCCCACTTTCTGTCTTTAAGAGGTTTAAAGAAACTACCAGACCATTTTCCAATGAATGTC TTGGTACCACCAGACCCGTAGTTCCTATTGATTCATCAGATTTTGCATTGGATATTCGCATGCCTGGGGTTACACCTAAACAGTCCGATACATACTTCTG CATGTCTATGCGAATACCAGTGGATGAGGAAGCCTTCGTGATTGACTTCAAGCCTCGAGCCAGCATGGATACTGTCCATCACATGTTACTTTTTGGATGC AATATGCCTTCATCCACTGGAAGTTACTGGTTTTGTGATGAAGGAACCTGTACAGATAAAGCCAATATTCTGTATGCCTGGGCGAGAAATGCTCCCCCTA CCCGGCTCCCCAAAGGTGTTGGATTCAGAGTTGGAGGAGAGACTGGAAGTAAATACTTTGTACTACAGGTACACTATGGGGATATTAGTGCTTTTAGAGA TAATAACAAGGACTGTTCTGGTGTGTCCTTACACCTCACACGTCTGCCACAGCCTTTAATTGCTGGCATGTACCTTATGATGTCTGTTGACACTGTTATC CCAGCAGGAGAAAAAGTGGTGAATTCTGACATTTCATGCCATTATAAAAATTATCCAATGCATGTCTTTGCCTATAGAGTTCACACTCACCATTTAGGTA AGGTAGTAAGTGGATACAGAGTAAGAAATGGACAGTGGACACTGATTGGACGGCAGAGCCCTCAGCTGCCACAGGCTTTCTACCCTGTGGGGCATCCAGT TGATGTAAGTTTTGGTGACCTACTGGCTGCAAGATGTGTATTCACTGGTGAAGGAAGGACAGAAGCCACACACATTGGTGGCACGTCTAGTGATGAAATG TGCAACTTATACATTATGTATTACATGGAAGCCAAGCATGCAGTTTCTTTCATGACCTGTACCCAGAATGTAGCTCCAGATATGTTCAGAACCATACCAC CAGAGGCCAACATTCCAATTCCCGTGAAGTCTGATATGGTTATGATGCATGAACATCATAAAGAAACAGAATATAAAGATAAGATTCCTTTACTACAGCA GCCAAAACGAGAAGAAGAAGAAGTGTTAGACCAGGATTTCCACATGGAAGAGGCACTGGATTGGCCTGGAGTATACTTGTTACCAGGCCAGGTTTCTGGG GTGGCTCTAGACCCTAAGAATAACCTGGTGATTTTCCACAGAGGTGACCATGTCTGGGATGGAAACTCGTTTGACAGCAAGTTTGTTTACCAGCAAATAG GACTCGGACCAATTGAAGAAGACACTATTCTTGTCATAGATCCAAATAATGCTGCAGTACTCCAGTCCAGTGGAAAAAATCTGTTTTACTTGCCACATGG CTTGAGTATAGATAAAGATGGGAATTATTGGGTCACAGACGTGGCTCTCCATCAGGTGTTCAAACTGGATCCAAACAATAAAGAAGGCCCTGTATTAATC CTGGGAAGGAGCATGCAACCAGGCAGTGACCAGAATCACTTCTGTCAACCCACTGATGTGGCTGTGGATCCAGGCACTGGAGCCATTTATGTATCAGATG GTTACTGCAACAGCAGGATTGTGCAGTTTTCACCAAGTGGAAAGTTCATCACACAGTGGGGAGAAGAGTCTTCAGGGAGCAGTCCTCTGCCAGGCCAGTT CACTGTTCCTCACAGCTTGGCTCTTGTGCCTCTTTTGGGCCAATTATGTGTGGCAGACCGGGAAAATGGTCGGATCCAGTGTTTTAAAACTGACACCAAA GAATTTGTGAGAGAGATTAAGCATTCATCATTTGGAAGAAATGTATTTGCAATTTCATATATACCAGGCTTGCTCTTTGCAGTGAATGGGAAGCCTCATT TTGGGGACCAAGAACCTGTACAAGGATTTGTGATGAACTTTTCCAATGGGGAAATTATAGACATCTTCAAGCCAGTGCGCAAGCACTTTGATATGCCTCA TGATATTGTTGCATCTGAAGATGGGACTGTGTACATTGGAGATGCTCATACCAACACCGTGTGGAAGTTCACCTTGACTGAGAAATTGGAACATCGATCA GTTAAAAAGGCTGGCATTGAGGTCCAGGAAATCAAAGAAGCCGAGGCAGTTGTTGAAACCAAAATGGAGAACAAACCCACCTCCTCAGAATTGCAGAAGA TGCAAGAGAAACAGAAACTGATCAAAGAGCCAGGCTCGGGAGTGCCTGTTGTTCTCATTACAACCCTTCTGGTTATTCCGGTGGTTGTCCTGCTGGCCAT TGCCATATTTATTCGGTGGAAAAAATCAAGGGCCTTTGGAGATTCTGAACACAAACTCGAGACGAGTTCAGGAAGAGTACTGGGAAGATTTAGAGGAAAG GGAAGTGGAGGCTTAAACCTTGGTAATTTCTTTGCAAGCCGTAAGGGCTACAGTCGAAAAGGGTTTGACCGGCTTAGCACTGAGGGCAGTGACCAAGAGA AAGAGGATGATGGAAGTGAATCAGAAGAGGAGTATTCAGCACCTCTGCCTGCGCTCGCACCTTCCTCCTCCTGAAAACCAAGCTTTGATTTAGATTGAGT AAGATTTACCCAGAATGTCAGATTCCTTTCCCTTTAGCACGTTTAAAGTTCTGTGTATTTAATTGTAAACTGTACTAGTCTGTGTGGGACTGTACACACT TTATTTACTTCGTTTTGGTTAAGTTGGCTTCTGTTTCTAGTTGAGGAGTTTCCTAAAAGTTCATAACAGTGCCATTGTCTTTATATGAACATAGACTAGA GAAACCGTCCTCTTTTTCCATCATAATTCTAATCTAACAATGGAAGATTTGCCCATTTACACTTTTGAGACTTTTTGGTGGATGTAAATAACCCCATTCT TTGCTTGAACACAGTATTTTCCCAATAGCACTTTCATTGCCAGTGTCTTTCTTTGGTGCCTTTCCTGTTCAGCATTCTTAGCCTGTGGCAGTAAAGAGAA ACTTTGTGCTACATGACGACAAAGCTGCTAAATCTCCTATTTTTTTAAAATCACTAACATTATATTGCAATGAAGGAAATAAAAAAGTCTCTATTTAAAT TCTTTTTTAAATTTTCTTCAGTTGGTGTGTTTTTGGGATGTCTTATTTTTAGATGGTTACACTGTTAGAACACTATTTTCAGAATCTGAATGTAATTTGT GTAATAAAGTGTTTTCAGAGCA

refseq_PAM.F1 (forward primer)
refseq_PAM.R1 (reverse primer)
Single exon skipping

Frame 0

VLNDD*RGFG*YPSQPLSFRSHKAPARLAPAPG HASGAGGRKASACGPDKSPACPRLFARRS*PRRL AAASSLLSLADGVWPPQDARRARAMK*RLLAAP LPNRQPQPRAALPGPLPAGSYRRGHGWPRP*PA SSPCFSKQLFGFPKPTFCL*EV*RNYQTIFQ*MS WYHQTRSSY*FIRFCIGYSHAWGYT*TVRYILL HVYANTSG*GSLRD*LQASSQHGYCPSHVTFWM QYAFIHWKLLVL**RNLYR*SQYSVCLGEKCSPY PAPQRCWIQSWRRDWK*ILCTTGTLWGY*CF*R **QGLFWCVLTPHTSATAFNCWHVPYDVC*HCY PSRRKSGEF*HFMPL*KLSNACLCL*SSHSPFR* GSKWIQSKKWTVDTDWTAEPSAATGFLPCGASS *CKFW*PTGCKMCIHW*RKDRSHTHWWHV***N VQLIHYVLHGSQACSFFHDLYPECSSRYVQNHTT RGQHSNSREV*YGYDA*TS*RNRI*R*DSFTTA AKTRRRRSVRPGFPHGRGTGLAWSILVTRPGFW GGSRP*E*PGDFPQR*PCLGWKLV*QQVCLPANR TRTN*RRHYSCHRSK*CCSTPVQWKKSVLLATW LEYR*RWELLGHRRGSPSGVQTGSKQ*RRPCIN PGKEHATRQ*PESLLSTH*CGCGSRHWSHLCIRW LLQQQDCAVFTKWKVHHTVGRRVFREQSSARPV HCSSQLGSCASFGPIMCGRPGKWSDPVF*N*HQ RICERD*AFIIWKKCICNFIYTRLALCSEWEASF WGPRTCTRICDELFQWGNYRHLQASAQAL*YAS *YCCI*RWDCVHWRCSYQHRVEVHLD*EIGTSI S*KGWH*GPGNQRSRGSC*NQNGEQTHLLRIAED ARETETDQRARLGSACCSHYNPSGYSGGCPAGH CHIYSVEKIKGLWRF*TQTRDEFRKSTGKI*RK GKWRLKPW*FLCKP*GLQSKRV*PA*H*GQ*PRE RG*WK*IRRGVFSTSACARTFLLLKTKL*FRLS KIYPECQIPFPLARLKFCVFNCKLY*SVWDCTH FIYFVLVKLASVSS*GVS*KFITVPLSLYEHRLE KPSSFSIIILI*QWKICPFTLLRLFGGCK*PHS LLEHSIFPIALSLPVSFFGAFPVQHS*PVAVKR NFVLHDDKAAKSPIFLKSLTLYCNEGNKKVSI*I LF*IFFSWCVFGMSYF*MVTLLEHYFQNLNVIC VIKCFQS

Frame 1

F*MMTDAGLGDTPHSPCHSGVIRHPRV*PQRQG TRAALEGGKLPPAGRTKVPPAHGFLPAARDRDA SPRPARCSRWRMVCGRRRTPAVPGP*SSGCWRRR CPTASPSPALRCPVLSRRGRIGVDMAGRVPSLL VLLVFPSSCLAFRSPLSVFKRFKETTRPFSNEC LGTTRPVVPIDSSDFALDIRMPGVTPKQSDTYFC MSMRIPVDEEAFVIDFKPRASMDTVHHMLLFGC NMPSSTGSYWFCDEGTCTDKANILYAWARNAPP TRLPKGVGFRVGGETGSKYFVLQVHYGDISAFRD NNKDCSGVSLHLTRLPQPLIAGMYLMMSVDTVI PAGEKVVNSDISCHYKNYPMHVFAYRVHTHHLG KVVSGYRVRNGQWTLIGRQSPQLPQAFYPVGHPV DVSFGDLLAARCVFTGEGRTEATHIGGTSSDEM CNLYIMYYMEAKHAVSFMTCTQNVAPDMFRTIP PEANIPIPVKSDMVMMHEHHKETEYKDKIPLLQQ PKREEEEVLDQDFHMEEALDWPGVYLLPGQVSG VALDPKNNLVIFHRGDHVWDGNSFDSKFVYQQI GLGPIEEDTILVIDPNNAAVLQSSGKNLFYLPHG LSIDKDGNYWVTDVALHQVFKLDPNNKEGPVLI LGRSMQPGSDQNHFCQPTDVAVDPGTGAIYVSD GYCNSRIVQFSPSGKFITQWGEESSGSSPLPGQF TVPHSLALVPLLGQLCVADRENGRIQCFKTDTK EFVREIKHSSFGRNVFAISYIPGLLFAVNGKPH FGDQEPVQGFVMNFSNGEIIDIFKPVRKHFDMPH DIVASEDGTVYIGDAHTNTVWKFTLTEKLEHRS VKKAGIEVQEIKEAEAVVETKMENKPTSSELQK MQEKQKLIKEPGSGVPVVLITTLLVIPVVVLLAI AIFIRWKKSRAFGDSEHKLETSSGRVLGRFRGK GSGGLNLGNFFASRKGYSRKGFDRLSTEGSDQE KEDDGSESEEEYSAPLPALAPSSS*KPSFDLD*V RFTQNVRFLSL*HV*SSVYLIVNCTSLCGTVHT LFTSFWLSWLLFLVEEFPKSS*QCHCLYMNID* RNRPLFPS*F*SNNGRFAHLHF*DFLVDVNNPIL CLNTVFSQ*HFHCQCLSLVPFLFSILSLWQ*RE TLCYMTTKLLNLLFF*NH*HYIAMKEIKKSLFK FFFKFSSVGVFLGCLIFRWLHC*NTIFRI*M*FV **SVFRA

Frame 2

SE**LTRVWVIPLTAPVIPES*GTRASSPSARA RERRWREESFRLRAGQKSRLPTAFCPPLVTETP RRGQLAALAGGWCVAAAGRPPCPGHEVAAAGGA AAQPPAPAPRCAARSSPGGVVSAWTWLAASLAC* FSLFFQAAVWLSEAHFLSLRGLKKLPDHFPMNV LVPPDP*FLLIHQILHWIFACLGLHLNSPIHTS ACLCEYQWMRKPS*LTSSLEPAWILSITCYFLDA ICLHPLEVTGFVMKEPVQIKPIFCMPGREMLPL PGSPKVLDSELEERLEVNTLYYRYTMGILVLLE IITRTVLVCPYTSHVCHSL*LLACTL*CLLTLLS QQEKKW*ILTFHAIIKIIQCMSLPIEFTLTI*V R**VDTE*EMDSGH*LDGRALSCHRLSTLWGIQ LM*VLVTYWLQDVYSLVKEGQKPHTLVARLVMKC ATYTLCITWKPSMQFLS*PVPRM*LQICSEPYH QRPTFQFP*SLIWL*CMNIIKKQNIKIRFLYYS SQNEKKKKC*TRISTWKRHWIGLEYTCYQARFLG WL*TLRITW*FSTEVTMSGMETRLTASLFTSK* DSDQLKKTLFLS*IQIMLQYSSPVEKICFTCHM A*V*IKMGIIGSQTWLSIRCSNWIQTIKKALY*S WEGACNQAVTRITSVNPLMWLWIQALEPFMYQM VTATAGLCSFHQVESSSHSGEKSLQGAVLCQAS SLFLTAWLLCLFWANYVWQTGKMVGSSVLKLTPK NL*ERLSIHHLEEMYLQFHIYQACSLQ*MGSLI LGTKNLYKDL**TFPMGKL*TSSSQCASTLICL MILLHLKMGLCTLEMLIPTPCGSSP*LRNWNIDQ LKRLALRSRKSKKPRQLLKPKWRTNPPPQNCRR CKRNRN*SKSQARECLLFSLQPFWLFRWLSCWP LPYLFGGKNQGPLEILNTNSRRVQEEYWEDLEER EVEA*TLVISLQAVRATVEKGLTGLALRAVTKR KRMMEVNQKRSIQHLCLRSHLPPPENQALI*IE *DLPRMSDSFPFSTFKVLCI*L*TVLVCVGLYTL YLLRFG*VGFCF*LRSFLKVHNSAIVFI*T*TR ETVLFFHHNSNLTMEDLPIYTFETFWWM*ITPF FA*TQYFPNSTFIASVFLWCLSCSAFLACGSKEK LCAT*RQSC*ISYFFKITNIILQ*RK*KSLYLN SFLNFLQLVCFWDVLFLDGYTVRTLFSESECNL CNKVFSE

Sequence NM_138766

GTTCTGAATGATGACTGACGCGGGTTTGGGTGATACCCCTCACAGCCCCTGTCATTCCGGAGTCATAAGGCACCCGCGCGTCTAGCCCCAGCGCCAGGGC ACGCGAGCGGCGCTGGAGGGAGGAAAGCTTCCGCCTGCGGGCCGGACAAAAGTCCCGCCTGCCCACGGCTTTTTGCCCGCCGCTCGTGACCGAGACGCCT CGCCGCGGCCAGCTCGCTGCTCTCGCTGGCGGATGGTGTGTGGCCGCCGCAGGACGCCCGCCGTGCCCGGGCCATGAAGTAGCGGCTGCTGGCGGCGCCG CTGCCCAACCGCCAGCCCCAGCCCCGCGCTGCGCTGCCCGGTCCTCTCCCGGCGGGGTCGTATCGGCGTGGACATGGCTGGCCGCGTCCCTAGCCTGCTA GTTCTCCTTGTTTTTCCAAGCAGCTGTTTGGCTTTCCGAAGCCCACTTTCTGTCTTTAAGAGGTTTAAAGAAACTACCAGACCATTTTCCAATGAATGTC TTGGTACCACCAGACCCGTAGTTCCTATTGATTCATCAGATTTTGCATTGGATATTCGCATGCCTGGGGTTACACCTAAACAGTCCGATACATACTTCTG CATGTCTATGCGAATACCAGTGGATGAGGAAGCCTTCGTGATTGACTTCAAGCCTCGAGCCAGCATGGATACTGTCCATCACATGTTACTTTTTGGATGC AATATGCCTTCATCCACTGGAAGTTACTGGTTTTGTGATGAAGGAACCTGTACAGATAAAGCCAATATTCTGTATGCCTGGGCGAGAAATGCTCCCCCTA CCCGGCTCCCCAAAGGTGTTGGATTCAGAGTTGGAGGAGAGACTGGAAGTAAATACTTTGTACTACAGGTACACTATGGGGATATTAGTGCTTTTAGAGA TAATAACAAGGACTGTTCTGGTGTGTCCTTACACCTCACACGTCTGCCACAGCCTTTAATTGCTGGCATGTACCTTATGATGTCTGTTGACACTGTTATC CCAGCAGGAGAAAAAGTGGTGAATTCTGACATTTCATGCCATTATAAAAATTATCCAATGCATGTCTTTGCCTATAGAGTTCACACTCACCATTTAGGTA AGGTAGTAAGTGGATACAGAGTAAGAAATGGACAGTGGACACTGATTGGACGGCAGAGCCCTCAGCTGCCACAGGCTTTCTACCCTGTGGGGCATCCAGT TGATGTAAGTTTTGGTGACCTACTGGCTGCAAGATGTGTATTCACTGGTGAAGGAAGGACAGAAGCCACACACATTGGTGGCACGTCTAGTGATGAAATG TGCAACTTATACATTATGTATTACATGGAAGCCAAGCATGCAGTTTCTTTCATGACCTGTACCCAGAATGTAGCTCCAGATATGTTCAGAACCATACCAC CAGAGGCCAACATTCCAATTCCCGTGAAGTCTGATATGGTTATGATGCATGAACATCATAAAGAAACAGAATATAAAGATAAGATTCCTTTACTACAGCA GCCAAAACGAGAAGAAGAAGAAGTGTTAGACCAGGGTGATTTCTATTCACTACTTTCCAAGCTGCTAGGAGAAAGGGAAGATGTTGTTCATGTGCACAAA TATAATCCTACAGAAAAGGCAGAATCAGAGTCAGACCTGGTAGCTGAGATTGCAAATGTAGTCCAAAAAAAGGATCTTGGTCGATCTGATGCCAGAGAGG GTGCAGAACATGAGAGGGGTAATGCTATTCTTGTCAGAGACAGAATTCACAAATTCCACAGACTAGTATCTACCTTGAGGCCACCAGAGAGCAGAGTTTT CTCATTACAGCAGCCCCCACCTGGTGAAGGCACCTGGGAACCAGAACACACAGGAGATTTCCACATGGAAGAGGCACTGGATTGGCCTGGAGTATACTTG TTACCAGGCCAGGTTTCTGGGGTGGCTCTAGACCCTAAGAATAACCTGGTGATTTTCCACAGAGGTGACCATGTCTGGGATGGAAACTCGTTTGACAGCA AGTTTGTTTACCAGCAAATAGGACTCGGACCAATTGAAGAAGACACTATTCTTGTCATAGATCCAAATAATGCTGCAGTACTCCAGTCCAGTGGAAAAAA TCTGTTTTACTTGCCACATGGCTTGAGTATAGATAAAGATGGGAATTATTGGGTCACAGACGTGGCTCTCCATCAGGTGTTCAAACTGGATCCAAACAAT AAAGAAGGCCCTGTATTAATCCTGGGAAGGAGCATGCAACCAGGCAGTGACCAGAATCACTTCTGTCAACCCACTGATGTGGCTGTGGATCCAGGCACTG GAGCCATTTATGTATCAGATGGTTACTGCAACAGCAGGATTGTGCAGTTTTCACCAAGTGGAAAGTTCATCACACAGTGGGGAGAAGAGTCTTCAGGGAG CAGTCCTCTGCCAGGCCAGTTCACTGTTCCTCACAGCTTGGCTCTTGTGCCTCTTTTGGGCCAATTATGTGTGGCAGACCGGGAAAATGGTCGGATCCAG TGTTTTAAAACTGACACCAAAGAATTTGTGAGAGAGATTAAGCATTCATCATTTGGAAGAAATGTATTTGCAATTTCATATATACCAGGCTTGCTCTTTG CAGTGAATGGGAAGCCTCATTTTGGGGACCAAGAACCTGTACAAGGATTTGTGATGAACTTTTCCAATGGGGAAATTATAGACATCTTCAAGCCAGTGCG CAAGCACTTTGATATGCCTCATGATATTGTTGCATCTGAAGATGGGACTGTGTACATTGGAGATGCTCATACCAACACCGTGTGGAAGTTCACCTTGACT GAGAAATTGGAACATCGATCAGTTAAAAAGGCTGGCATTGAGGTCCAGGAAATCAAAGATTCTGAACACAAACTCGAGACGAGTTCAGGAAGAGTACTGG GAAGATTTAGAGGAAAGGGAAGTGGAGGCTTAAACCTTGGTAATTTCTTTGCAAGCCGTAAGGGCTACAGTCGAAAAGGGTTTGACCGGCTTAGCACTGA GGGCAGTGACCAAGAGAAAGAGGATGATGGAAGTGAATCAGAAGAGGAGTATTCAGCACCTCTGCCTGCGCTCGCACCTTCCTCCTCCTGAAAACCAAGC TTTGATTTAGATTGAGTAAGATTTACCCAGAATGTCAGATTCCTTTCCCTTTAGCACGTTTAAAGTTCTGTGTATTTAATTGTAAACTGTACTAGTCTGT GTGGGACTGTACACACTTTATTTACTTCGTTTTGGTTAAGTTGGCTTCTGTTTCTAGTTGAGGAGTTTCCTAAAAGTTCATAACAGTGCCATTGTCTTTA TATGAACATAGACTAGAGAAACCGTCCTCTTTTTCCATCATAATTCTAATCTAACAATGGAAGATTTGCCCATTTACACTTTTGAGACTTTTTGGTGGAT GTAAATAACCCCATTCTTTGCTTGAACACAGTATTTTCCCAATAGCACTTTCATTGCCAGTGTCTTTCTTTGGTGCCTTTCCTGTTCAGCATTCTTAGCC TGTGGCAGTAAAGAGAAACTTTGTGCTACATGACGACAAAGCTGCTAAATCTCCTATTTTTTTAAAATCACTAACATTATATTGCAATGAAGGAAATAAA AAAGTCTCTATTTAAATTCTTTTTTAAATTTTCTTCAGTTGGTGTGTTTTTGGGATGTCTTATTTTTAGATGGTTACACTGTTAGAACACTATTTTCAGA ATCTGAATGTAATTTGTGTAATAAAGTGTTTTCAGAGCA

refseq_PAM.F1 (forward primer)
refseq_PAM.R1 (reverse primer)
Single exon skipping

Frame 0

VLNDD*RGFG*YPSQPLSFRSHKAPARLAPAPG HASGAGGRKASACGPDKSPACPRLFARRS*PRRL AAASSLLSLADGVWPPQDARRARAMK*RLLAAP LPNRQPQPRAALPGPLPAGSYRRGHGWPRP*PA SSPCFSKQLFGFPKPTFCL*EV*RNYQTIFQ*MS WYHQTRSSY*FIRFCIGYSHAWGYT*TVRYILL HVYANTSG*GSLRD*LQASSQHGYCPSHVTFWM QYAFIHWKLLVL**RNLYR*SQYSVCLGEKCSPY PAPQRCWIQSWRRDWK*ILCTTGTLWGY*CF*R **QGLFWCVLTPHTSATAFNCWHVPYDVC*HCY PSRRKSGEF*HFMPL*KLSNACLCL*SSHSPFR* GSKWIQSKKWTVDTDWTAEPSAATGFLPCGASS *CKFW*PTGCKMCIHW*RKDRSHTHWWHV***N VQLIHYVLHGSQACSFFHDLYPECSSRYVQNHTT RGQHSNSREV*YGYDA*TS*RNRI*R*DSFTTA AKTRRRRSVRPG*FLFTTFQAARRKGRCCSCAQ I*SYRKGRIRVRPGS*DCKCSPKKGSWSI*CQRG CRT*EG*CYSCQRQNSQIPQTSIYLEATREQSF LITAAPTW*RHLGTRTHRRFPHGRGTGLAWSIL VTRPGFWGGSRP*E*PGDFPQR*PCLGWKLV*QQ VCLPANRTRTN*RRHYSCHRSK*CCSTPVQWKK SVLLATWLEYR*RWELLGHRRGSPSGVQTGSKQ *RRPCINPGKEHATRQ*PESLLSTH*CGCGSRHW SHLCIRWLLQQQDCAVFTKWKVHHTVGRRVFRE QSSARPVHCSSQLGSCASFGPIMCGRPGKWSDP VF*N*HQRICERD*AFIIWKKCICNFIYTRLALC SEWEASFWGPRTCTRICDELFQWGNYRHLQASA QAL*YAS*YCCI*RWDCVHWRCSYQHRVEVHLD *EIGTSIS*KGWH*GPGNQRF*TQTRDEFRKSTG KI*RKGKWRLKPW*FLCKP*GLQSKRV*PA*H* GQ*PRERG*WK*IRRGVFSTSACARTFLLLKTK L*FRLSKIYPECQIPFPLARLKFCVFNCKLY*SV WDCTHFIYFVLVKLASVSS*GVS*KFITVPLSL YEHRLEKPSSFSIIILI*QWKICPFTLLRLFGG CK*PHSLLEHSIFPIALSLPVSFFGAFPVQHS*P VAVKRNFVLHDDKAAKSPIFLKSLTLYCNEGNK KVSI*ILF*IFFSWCVFGMSYF*MVTLLEHYFQ NLNVICVIKCFQS

Frame 1

F*MMTDAGLGDTPHSPCHSGVIRHPRV*PQRQG TRAALEGGKLPPAGRTKVPPAHGFLPAARDRDA SPRPARCSRWRMVCGRRRTPAVPGP*SSGCWRRR CPTASPSPALRCPVLSRRGRIGVDMAGRVPSLL VLLVFPSSCLAFRSPLSVFKRFKETTRPFSNEC LGTTRPVVPIDSSDFALDIRMPGVTPKQSDTYFC MSMRIPVDEEAFVIDFKPRASMDTVHHMLLFGC NMPSSTGSYWFCDEGTCTDKANILYAWARNAPP TRLPKGVGFRVGGETGSKYFVLQVHYGDISAFRD NNKDCSGVSLHLTRLPQPLIAGMYLMMSVDTVI PAGEKVVNSDISCHYKNYPMHVFAYRVHTHHLG KVVSGYRVRNGQWTLIGRQSPQLPQAFYPVGHPV DVSFGDLLAARCVFTGEGRTEATHIGGTSSDEM CNLYIMYYMEAKHAVSFMTCTQNVAPDMFRTIP PEANIPIPVKSDMVMMHEHHKETEYKDKIPLLQQ PKREEEEVLDQGDFYSLLSKLLGEREDVVHVHK YNPTEKAESESDLVAEIANVVQKKDLGRSDARE GAEHERGNAILVRDRIHKFHRLVSTLRPPESRVF SLQQPPPGEGTWEPEHTGDFHMEEALDWPGVYL LPGQVSGVALDPKNNLVIFHRGDHVWDGNSFDS KFVYQQIGLGPIEEDTILVIDPNNAAVLQSSGKN LFYLPHGLSIDKDGNYWVTDVALHQVFKLDPNN KEGPVLILGRSMQPGSDQNHFCQPTDVAVDPGT GAIYVSDGYCNSRIVQFSPSGKFITQWGEESSGS SPLPGQFTVPHSLALVPLLGQLCVADRENGRIQ CFKTDTKEFVREIKHSSFGRNVFAISYIPGLLF AVNGKPHFGDQEPVQGFVMNFSNGEIIDIFKPVR KHFDMPHDIVASEDGTVYIGDAHTNTVWKFTLT EKLEHRSVKKAGIEVQEIKDSEHKLETSSGRVL GRFRGKGSGGLNLGNFFASRKGYSRKGFDRLSTE GSDQEKEDDGSESEEEYSAPLPALAPSSS*KPS FDLD*VRFTQNVRFLSL*HV*SSVYLIVNCTSL CGTVHTLFTSFWLSWLLFLVEEFPKSS*QCHCLY MNID*RNRPLFPS*F*SNNGRFAHLHF*DFLVD VNNPILCLNTVFSQ*HFHCQCLSLVPFLFSILS LWQ*RETLCYMTTKLLNLLFF*NH*HYIAMKEIK KSLFKFFFKFSSVGVFLGCLIFRWLHC*NTIFR I*M*FV**SVFRA

Frame 2

SE**LTRVWVIPLTAPVIPES*GTRASSPSARA RERRWREESFRLRAGQKSRLPTAFCPPLVTETP RRGQLAALAGGWCVAAAGRPPCPGHEVAAAGGA AAQPPAPAPRCAARSSPGGVVSAWTWLAASLAC* FSLFFQAAVWLSEAHFLSLRGLKKLPDHFPMNV LVPPDP*FLLIHQILHWIFACLGLHLNSPIHTS ACLCEYQWMRKPS*LTSSLEPAWILSITCYFLDA ICLHPLEVTGFVMKEPVQIKPIFCMPGREMLPL PGSPKVLDSELEERLEVNTLYYRYTMGILVLLE IITRTVLVCPYTSHVCHSL*LLACTL*CLLTLLS QQEKKW*ILTFHAIIKIIQCMSLPIEFTLTI*V R**VDTE*EMDSGH*LDGRALSCHRLSTLWGIQ LM*VLVTYWLQDVYSLVKEGQKPHTLVARLVMKC ATYTLCITWKPSMQFLS*PVPRM*LQICSEPYH QRPTFQFP*SLIWL*CMNIIKKQNIKIRFLYYS SQNEKKKKC*TRVISIHYFPSC*EKGKMLFMCTN IILQKRQNQSQTW*LRLQM*SKKRILVDLMPER VQNMRGVMLFLSETEFTNSTD*YLP*GHQRAEF SHYSSPHLVKAPGNQNTQEISTWKRHWIGLEYTC YQARFLGWL*TLRITW*FSTEVTMSGMETRLTA SLFTSK*DSDQLKKTLFLS*IQIMLQYSSPVEK ICFTCHMA*V*IKMGIIGSQTWLSIRCSNWIQTI KKALY*SWEGACNQAVTRITSVNPLMWLWIQAL EPFMYQMVTATAGLCSFHQVESSSHSGEKSLQG AVLCQASSLFLTAWLLCLFWANYVWQTGKMVGSS VLKLTPKNL*ERLSIHHLEEMYLQFHIYQACSL Q*MGSLILGTKNLYKDL**TFPMGKL*TSSSQC ASTLICLMILLHLKMGLCTLEMLIPTPCGSSP*L RNWNIDQLKRLALRSRKSKILNTNSRRVQEEYW EDLEEREVEA*TLVISLQAVRATVEKGLTGLAL RAVTKRKRMMEVNQKRSIQHLCLRSHLPPPENQA LI*IE*DLPRMSDSFPFSTFKVLCI*L*TVLVC VGLYTLYLLRFG*VGFCF*LRSFLKVHNSAIVF I*T*TRETVLFFHHNSNLTMEDLPIYTFETFWWM *ITPFFA*TQYFPNSTFIASVFLWCLSCSAFLA CGSKEKLCAT*RQSC*ISYFFKITNIILQ*RK* KSLYLNSFLNFLQLVCFWDVLFLDGYTVRTLFSE SECNLCNKVFSE

Sequence NM_000919

GTTCTGAATGATGACTGACGCGGGTTTGGGTGATACCCCTCACAGCCCCTGTCATTCCGGAGTCATAAGGCACCCGCGCGTCTAGCCCCAGCGCCAGGGC ACGCGAGCGGCGCTGGAGGGAGGAAAGCTTCCGCCTGCGGGCCGGACAAAAGTCCCGCCTGCCCACGGCTTTTTGCCCGCCGCTCGTGACCGAGACGCCT CGCCGCGGCCAGCTCGCTGCTCTCGCTGGCGGATGGTGTGTGGCCGCCGCAGGACGCCCGCCGTGCCCGGGCCATGAAGTAGCGGCTGCTGGCGGCGCCG CTGCCCAACCGCCAGCCCCAGCCCCGCGCTGCGCTGCCCGGTCCTCTCCCGGCGGGGTCGTATCGGCGTGGACATGGCTGGCCGCGTCCCTAGCCTGCTA GTTCTCCTTGTTTTTCCAAGCAGCTGTTTGGCTTTCCGAAGCCCACTTTCTGTCTTTAAGAGGTTTAAAGAAACTACCAGACCATTTTCCAATGAATGTC TTGGTACCACCAGACCCGTAGTTCCTATTGATTCATCAGATTTTGCATTGGATATTCGCATGCCTGGGGTTACACCTAAACAGTCCGATACATACTTCTG CATGTCTATGCGAATACCAGTGGATGAGGAAGCCTTCGTGATTGACTTCAAGCCTCGAGCCAGCATGGATACTGTCCATCACATGTTACTTTTTGGATGC AATATGCCTTCATCCACTGGAAGTTACTGGTTTTGTGATGAAGGAACCTGTACAGATAAAGCCAATATTCTGTATGCCTGGGCGAGAAATGCTCCCCCTA CCCGGCTCCCCAAAGGTGTTGGATTCAGAGTTGGAGGAGAGACTGGAAGTAAATACTTTGTACTACAGGTACACTATGGGGATATTAGTGCTTTTAGAGA TAATAACAAGGACTGTTCTGGTGTGTCCTTACACCTCACACGTCTGCCACAGCCTTTAATTGCTGGCATGTACCTTATGATGTCTGTTGACACTGTTATC CCAGCAGGAGAAAAAGTGGTGAATTCTGACATTTCATGCCATTATAAAAATTATCCAATGCATGTCTTTGCCTATAGAGTTCACACTCACCATTTAGGTA AGGTAGTAAGTGGATACAGAGTAAGAAATGGACAGTGGACACTGATTGGACGGCAGAGCCCTCAGCTGCCACAGGCTTTCTACCCTGTGGGGCATCCAGT TGATGTAAGTTTTGGTGACCTACTGGCTGCAAGATGTGTATTCACTGGTGAAGGAAGGACAGAAGCCACACACATTGGTGGCACGTCTAGTGATGAAATG TGCAACTTATACATTATGTATTACATGGAAGCCAAGCATGCAGTTTCTTTCATGACCTGTACCCAGAATGTAGCTCCAGATATGTTCAGAACCATACCAC CAGAGGCCAACATTCCAATTCCCGTGAAGTCTGATATGGTTATGATGCATGAACATCATAAAGAAACAGAATATAAAGATAAGATTCCTTTACTACAGCA GCCAAAACGAGAAGAAGAAGAAGTGTTAGACCAGGGTGATTTCTATTCACTACTTTCCAAGCTGCTAGGAGAAAGGGAAGATGTTGTTCATGTGCACAAA TATAATCCTACAGAAAAGGCAGAATCAGAGTCAGACCTGGTAGCTGAGATTGCAAATGTAGTCCAAAAAAAGGATCTTGGTCGATCTGATGCCAGAGAGG GTGCAGAACATGAGAGGGGTAATGCTATTCTTGTCAGAGACAGAATTCACAAATTCCACAGACTAGTATCTACCTTGAGGCCACCAGAGAGCAGAGTTTT CTCATTACAGCAGCCCCCACCTGGTGAAGGCACCTGGGAACCAGAACACACAGGAGATTTCCACATGGAAGAGGCACTGGATTGGCCTGGAGTATACTTG TTACCAGGCCAGGTTTCTGGGGTGGCTCTAGACCCTAAGAATAACCTGGTGATTTTCCACAGAGGTGACCATGTCTGGGATGGAAACTCGTTTGACAGCA AGTTTGTTTACCAGCAAATAGGACTCGGACCAATTGAAGAAGACACTATTCTTGTCATAGATCCAAATAATGCTGCAGTACTCCAGTCCAGTGGAAAAAA TCTGTTTTACTTGCCACATGGCTTGAGTATAGATAAAGATGGGAATTATTGGGTCACAGACGTGGCTCTCCATCAGGTGTTCAAACTGGATCCAAACAAT AAAGAAGGCCCTGTATTAATCCTGGGAAGGAGCATGCAACCAGGCAGTGACCAGAATCACTTCTGTCAACCCACTGATGTGGCTGTGGATCCAGGCACTG GAGCCATTTATGTATCAGATGGTTACTGCAACAGCAGGATTGTGCAGTTTTCACCAAGTGGAAAGTTCATCACACAGTGGGGAGAAGAGTCTTCAGGGAG CAGTCCTCTGCCAGGCCAGTTCACTGTTCCTCACAGCTTGGCTCTTGTGCCTCTTTTGGGCCAATTATGTGTGGCAGACCGGGAAAATGGTCGGATCCAG TGTTTTAAAACTGACACCAAAGAATTTGTGAGAGAGATTAAGCATTCATCATTTGGAAGAAATGTATTTGCAATTTCATATATACCAGGCTTGCTCTTTG CAGTGAATGGGAAGCCTCATTTTGGGGACCAAGAACCTGTACAAGGATTTGTGATGAACTTTTCCAATGGGGAAATTATAGACATCTTCAAGCCAGTGCG CAAGCACTTTGATATGCCTCATGATATTGTTGCATCTGAAGATGGGACTGTGTACATTGGAGATGCTCATACCAACACCGTGTGGAAGTTCACCTTGACT GAGAAATTGGAACATCGATCAGTTAAAAAGGCTGGCATTGAGGTCCAGGAAATCAAAGAAGCCGAGGCAGTTGTTGAAACCAAAATGGAGAACAAACCCA CCTCCTCAGAATTGCAGAAGATGCAAGAGAAACAGAAACTGATCAAAGAGCCAGGCTCGGGAGTGCCTGTTGTTCTCATTACAACCCTTCTGGTTATTCC GGTGGTTGTCCTGCTGGCCATTGCCATATTTATTCGGTGGAAAAAATCAAGGGCCTTTGGAGCAGATTCTGAACACAAACTCGAGACGAGTTCAGGAAGA GTACTGGGAAGATTTAGAGGAAAGGGAAGTGGAGGCTTAAACCTTGGTAATTTCTTTGCAAGCCGTAAGGGCTACAGTCGAAAAGGGTTTGACCGGCTTA GCACTGAGGGCAGTGACCAAGAGAAAGAGGATGATGGAAGTGAATCAGAAGAGGAGTATTCAGCACCTCTGCCTGCGCTCGCACCTTCCTCCTCCTGAAA ACCAAGCTTTGATTTAGATTGAGTAAGATTTACCCAGAATGTCAGATTCCTTTCCCTTTAGCACGTTTAAAGTTCTGTGTATTTAATTGTAAACTGTACT AGTCTGTGTGGGACTGTACACACTTTATTTACTTCGTTTTGGTTAAGTTGGCTTCTGTTTCTAGTTGAGGAGTTTCCTAAAAGTTCATAACAGTGCCATT GTCTTTATATGAACATAGACTAGAGAAACCGTCCTCTTTTTCCATCATAATTCTAATCTAACAATGGAAGATTTGCCCATTTACACTTTTGAGACTTTTT GGTGGATGTAAATAACCCCATTCTTTGCTTGAACACAGTATTTTCCCAATAGCACTTTCATTGCCAGTGTCTTTCTTTGGTGCCTTTCCTGTTCAGCATT CTTAGCCTGTGGCAGTAAAGAGAAACTTTGTGCTACATGACGACAAAGCTGCTAAATCTCCTATTTTTTTAAAATCACTAACATTATATTGCAATGAAGG AAATAAAAAAGTCTCTATTTAAATTCTTTTTTAAATTTTCTTCAGTTGGTGTGTTTTTGGGATGTCTTATTTTTAGATGGTTACACTGTTAGAACACTAT TTTCAGAATCTGAATGTAATTTGTGTAATAAAGTGTTTTCAGAGCA

refseq_PAM.F1 (forward primer)
refseq_PAM.R1 (reverse primer)
Single exon skipping

Frame 0

VLNDD*RGFG*YPSQPLSFRSHKAPARLAPAPG HASGAGGRKASACGPDKSPACPRLFARRS*PRRL AAASSLLSLADGVWPPQDARRARAMK*RLLAAP LPNRQPQPRAALPGPLPAGSYRRGHGWPRP*PA SSPCFSKQLFGFPKPTFCL*EV*RNYQTIFQ*MS WYHQTRSSY*FIRFCIGYSHAWGYT*TVRYILL HVYANTSG*GSLRD*LQASSQHGYCPSHVTFWM QYAFIHWKLLVL**RNLYR*SQYSVCLGEKCSPY PAPQRCWIQSWRRDWK*ILCTTGTLWGY*CF*R **QGLFWCVLTPHTSATAFNCWHVPYDVC*HCY PSRRKSGEF*HFMPL*KLSNACLCL*SSHSPFR* GSKWIQSKKWTVDTDWTAEPSAATGFLPCGASS *CKFW*PTGCKMCIHW*RKDRSHTHWWHV***N VQLIHYVLHGSQACSFFHDLYPECSSRYVQNHTT RGQHSNSREV*YGYDA*TS*RNRI*R*DSFTTA AKTRRRRSVRPG*FLFTTFQAARRKGRCCSCAQ I*SYRKGRIRVRPGS*DCKCSPKKGSWSI*CQRG CRT*EG*CYSCQRQNSQIPQTSIYLEATREQSF LITAAPTW*RHLGTRTHRRFPHGRGTGLAWSIL VTRPGFWGGSRP*E*PGDFPQR*PCLGWKLV*QQ VCLPANRTRTN*RRHYSCHRSK*CCSTPVQWKK SVLLATWLEYR*RWELLGHRRGSPSGVQTGSKQ *RRPCINPGKEHATRQ*PESLLSTH*CGCGSRHW SHLCIRWLLQQQDCAVFTKWKVHHTVGRRVFRE QSSARPVHCSSQLGSCASFGPIMCGRPGKWSDP VF*N*HQRICERD*AFIIWKKCICNFIYTRLALC SEWEASFWGPRTCTRICDELFQWGNYRHLQASA QAL*YAS*YCCI*RWDCVHWRCSYQHRVEVHLD *EIGTSIS*KGWH*GPGNQRSRGSC*NQNGEQTH LLRIAEDARETETDQRARLGSACCSHYNPSGYS GGCPAGHCHIYSVEKIKGLWSRF*TQTRDEFRK STGKI*RKGKWRLKPW*FLCKP*GLQSKRV*PA* H*GQ*PRERG*WK*IRRGVFSTSACARTFLLLK TKL*FRLSKIYPECQIPFPLARLKFCVFNCKLY *SVWDCTHFIYFVLVKLASVSS*GVS*KFITVPL SLYEHRLEKPSSFSIIILI*QWKICPFTLLRLF GGCK*PHSLLEHSIFPIALSLPVSFFGAFPVQH S*PVAVKRNFVLHDDKAAKSPIFLKSLTLYCNEG NKKVSI*ILF*IFFSWCVFGMSYF*MVTLLEHY FQNLNVICVIKCFQS

Frame 1

F*MMTDAGLGDTPHSPCHSGVIRHPRV*PQRQG TRAALEGGKLPPAGRTKVPPAHGFLPAARDRDA SPRPARCSRWRMVCGRRRTPAVPGP*SSGCWRRR CPTASPSPALRCPVLSRRGRIGVDMAGRVPSLL VLLVFPSSCLAFRSPLSVFKRFKETTRPFSNEC LGTTRPVVPIDSSDFALDIRMPGVTPKQSDTYFC MSMRIPVDEEAFVIDFKPRASMDTVHHMLLFGC NMPSSTGSYWFCDEGTCTDKANILYAWARNAPP TRLPKGVGFRVGGETGSKYFVLQVHYGDISAFRD NNKDCSGVSLHLTRLPQPLIAGMYLMMSVDTVI PAGEKVVNSDISCHYKNYPMHVFAYRVHTHHLG KVVSGYRVRNGQWTLIGRQSPQLPQAFYPVGHPV DVSFGDLLAARCVFTGEGRTEATHIGGTSSDEM CNLYIMYYMEAKHAVSFMTCTQNVAPDMFRTIP PEANIPIPVKSDMVMMHEHHKETEYKDKIPLLQQ PKREEEEVLDQGDFYSLLSKLLGEREDVVHVHK YNPTEKAESESDLVAEIANVVQKKDLGRSDARE GAEHERGNAILVRDRIHKFHRLVSTLRPPESRVF SLQQPPPGEGTWEPEHTGDFHMEEALDWPGVYL LPGQVSGVALDPKNNLVIFHRGDHVWDGNSFDS KFVYQQIGLGPIEEDTILVIDPNNAAVLQSSGKN LFYLPHGLSIDKDGNYWVTDVALHQVFKLDPNN KEGPVLILGRSMQPGSDQNHFCQPTDVAVDPGT GAIYVSDGYCNSRIVQFSPSGKFITQWGEESSGS SPLPGQFTVPHSLALVPLLGQLCVADRENGRIQ CFKTDTKEFVREIKHSSFGRNVFAISYIPGLLF AVNGKPHFGDQEPVQGFVMNFSNGEIIDIFKPVR KHFDMPHDIVASEDGTVYIGDAHTNTVWKFTLT EKLEHRSVKKAGIEVQEIKEAEAVVETKMENKP TSSELQKMQEKQKLIKEPGSGVPVVLITTLLVIP VVVLLAIAIFIRWKKSRAFGADSEHKLETSSGR VLGRFRGKGSGGLNLGNFFASRKGYSRKGFDRL STEGSDQEKEDDGSESEEEYSAPLPALAPSSS*K PSFDLD*VRFTQNVRFLSL*HV*SSVYLIVNCT SLCGTVHTLFTSFWLSWLLFLVEEFPKSS*QCH CLYMNID*RNRPLFPS*F*SNNGRFAHLHF*DFL VDVNNPILCLNTVFSQ*HFHCQCLSLVPFLFSI LSLWQ*RETLCYMTTKLLNLLFF*NH*HYIAMK EIKKSLFKFFFKFSSVGVFLGCLIFRWLHC*NTI FRI*M*FV**SVFRA

Frame 2

SE**LTRVWVIPLTAPVIPES*GTRASSPSARA RERRWREESFRLRAGQKSRLPTAFCPPLVTETP RRGQLAALAGGWCVAAAGRPPCPGHEVAAAGGA AAQPPAPAPRCAARSSPGGVVSAWTWLAASLAC* FSLFFQAAVWLSEAHFLSLRGLKKLPDHFPMNV LVPPDP*FLLIHQILHWIFACLGLHLNSPIHTS ACLCEYQWMRKPS*LTSSLEPAWILSITCYFLDA ICLHPLEVTGFVMKEPVQIKPIFCMPGREMLPL PGSPKVLDSELEERLEVNTLYYRYTMGILVLLE IITRTVLVCPYTSHVCHSL*LLACTL*CLLTLLS QQEKKW*ILTFHAIIKIIQCMSLPIEFTLTI*V R**VDTE*EMDSGH*LDGRALSCHRLSTLWGIQ LM*VLVTYWLQDVYSLVKEGQKPHTLVARLVMKC ATYTLCITWKPSMQFLS*PVPRM*LQICSEPYH QRPTFQFP*SLIWL*CMNIIKKQNIKIRFLYYS SQNEKKKKC*TRVISIHYFPSC*EKGKMLFMCTN IILQKRQNQSQTW*LRLQM*SKKRILVDLMPER VQNMRGVMLFLSETEFTNSTD*YLP*GHQRAEF SHYSSPHLVKAPGNQNTQEISTWKRHWIGLEYTC YQARFLGWL*TLRITW*FSTEVTMSGMETRLTA SLFTSK*DSDQLKKTLFLS*IQIMLQYSSPVEK ICFTCHMA*V*IKMGIIGSQTWLSIRCSNWIQTI KKALY*SWEGACNQAVTRITSVNPLMWLWIQAL EPFMYQMVTATAGLCSFHQVESSSHSGEKSLQG AVLCQASSLFLTAWLLCLFWANYVWQTGKMVGSS VLKLTPKNL*ERLSIHHLEEMYLQFHIYQACSL Q*MGSLILGTKNLYKDL**TFPMGKL*TSSSQC ASTLICLMILLHLKMGLCTLEMLIPTPCGSSP*L RNWNIDQLKRLALRSRKSKKPRQLLKPKWRTNP PPQNCRRCKRNRN*SKSQARECLLFSLQPFWLF RWLSCWPLPYLFGGKNQGPLEQILNTNSRRVQEE YWEDLEEREVEA*TLVISLQAVRATVEKGLTGL ALRAVTKRKRMMEVNQKRSIQHLCLRSHLPPPE NQALI*IE*DLPRMSDSFPFSTFKVLCI*L*TVL VCVGLYTLYLLRFG*VGFCF*LRSFLKVHNSAI VFI*T*TRETVLFFHHNSNLTMEDLPIYTFETF WWM*ITPFFA*TQYFPNSTFIASVFLWCLSCSAF LACGSKEKLCAT*RQSC*ISYFFKITNIILQ*R K*KSLYLNSFLNFLQLVCFWDVLFLDGYTVRTL FSESECNLCNKVFSE

Sequence NM_138822

GTTCTGAATGATGACTGACGCGGGTTTGGGTGATACCCCTCACAGCCCCTGTCATTCCGGAGTCATAAGGCACCCGCGCGTCTAGCCCCAGCGCCAGGGC ACGCGAGCGGCGCTGGAGGGAGGAAAGCTTCCGCCTGCGGGCCGGACAAAAGTCCCGCCTGCCCACGGCTTTTTGCCCGCCGCTCGTGACCGAGACGCCT CGCCGCGGCCAGCTCGCTGCTCTCGCTGGCGGATGGTGTGTGGCCGCCGCAGGACGCCCGCCGTGCCCGGGCCATGAAGTAGCGGCTGCTGGCGGCGCCG CTGCCCAACCGCCAGCCCCAGCCCCGCGCTGCGCTGCCCGGTCCTCTCCCGGCGGGGTCGTATCGGCGTGGACATGGCTGGCCGCGTCCCTAGCCTGCTA GTTCTCCTTGTTTTTCCAAGCAGCTGTTTGGCTTTCCGAAGCCCACTTTCTGTCTTTAAGAGGTTTAAAGAAACTACCAGACCATTTTCCAATGAATGTC TTGGTACCACCAGACCCGTAGTTCCTATTGATTCATCAGATTTTGCATTGGATATTCGCATGCCTGGGGTTACACCTAAACAGTCCGATACATACTTCTG CATGTCTATGCGAATACCAGTGGATGAGGAAGCCTTCGTGATTGACTTCAAGCCTCGAGCCAGCATGGATACTGTCCATCACATGTTACTTTTTGGATGC AATATGCCTTCATCCACTGGAAGTTACTGGTTTTGTGATGAAGGAACCTGTACAGATAAAGCCAATATTCTGTATGCCTGGGCGAGAAATGCTCCCCCTA CCCGGCTCCCCAAAGGTGTTGGATTCAGAGTTGGAGGAGAGACTGGAAGTAAATACTTTGTACTACAGGTACACTATGGGGATATTAGTGCTTTTAGAGA TAATAACAAGGACTGTTCTGGTGTGTCCTTACACCTCACACGTCTGCCACAGCCTTTAATTGCTGGCATGTACCTTATGATGTCTGTTGACACTGTTATC CCAGCAGGAGAAAAAGTGGTGAATTCTGACATTTCATGCCATTATAAAAATTATCCAATGCATGTCTTTGCCTATAGAGTTCACACTCACCATTTAGGTA AGGTAGTAAGTGGATACAGAGTAAGAAATGGACAGTGGACACTGATTGGACGGCAGAGCCCTCAGCTGCCACAGGCTTTCTACCCTGTGGGGCATCCAGT TGATGTAAGTTTTGGTGACCTACTGGCTGCAAGATGTGTATTCACTGGTGAAGGAAGGACAGAAGCCACACACATTGGTGGCACGTCTAGTGATGAAATG TGCAACTTATACATTATGTATTACATGGAAGCCAAGCATGCAGTTTCTTTCATGACCTGTACCCAGAATGTAGCTCCAGATATGTTCAGAACCATACCAC CAGAGGCCAACATTCCAATTCCCGTGAAGTCTGATATGGTTATGATGCATGAACATCATAAAGAAACAGAATATAAAGATAAGATTCCTTTACTACAGCA GCCAAAACGAGAAGAAGAAGAAGTGTTAGACCAGGGTGATTTCTATTCACTACTTTCCAAGCTGCTAGGAGAAAGGGAAGATGTTGTTCATGTGCACAAA TATAATCCTACAGAAAAGGCAGAATCAGAGTCAGACCTGGTAGCTGAGATTGCAAATGTAGTCCAAAAAAAGGATCTTGGTCGATCTGATGCCAGAGAGG GTGCAGAACATGAGAGGGGTAATGCTATTCTTGTCAGAGACAGAATTCACAAATTCCACAGACTAGTATCTACCTTGAGGCCACCAGAGAGCAGAGTTTT CTCATTACAGCAGCCCCCACCTGGTGAAGGCACCTGGGAACCAGAACACACAGGAGATTTCCACATGGAAGAGGCACTGGATTGGCCTGGAGTATACTTG TTACCAGGCCAGGTTTCTGGGGTGGCTCTAGACCCTAAGAATAACCTGGTGATTTTCCACAGAGGTGACCATGTCTGGGATGGAAACTCGTTTGACAGCA AGTTTGTTTACCAGCAAATAGGACTCGGACCAATTGAAGAAGACACTATTCTTGTCATAGATCCAAATAATGCTGCAGTACTCCAGTCCAGTGGAAAAAA TCTGTTTTACTTGCCACATGGCTTGAGTATAGATAAAGATGGGAATTATTGGGTCACAGACGTGGCTCTCCATCAGGTGTTCAAACTGGATCCAAACAAT AAAGAAGGCCCTGTATTAATCCTGGGAAGGAGCATGCAACCAGGCAGTGACCAGAATCACTTCTGTCAACCCACTGATGTGGCTGTGGATCCAGGCACTG GAGCCATTTATGTATCAGATGGTTACTGCAACAGCAGGATTGTGCAGTTTTCACCAAGTGGAAAGTTCATCACACAGTGGGGAGAAGAGTCTTCAGGGAG CAGTCCTCTGCCAGGCCAGTTCACTGTTCCTCACAGCTTGGCTCTTGTGCCTCTTTTGGGCCAATTATGTGTGGCAGACCGGGAAAATGGTCGGATCCAG TGTTTTAAAACTGACACCAAAGAATTTGTGAGAGAGATTAAGCATTCATCATTTGGAAGAAATGTATTTGCAATTTCATATATACCAGGCTTGCTCTTTG CAGTGAATGGGAAGCCTCATTTTGGGGACCAAGAACCTGTACAAGGATTTGTGATGAACTTTTCCAATGGGGAAATTATAGACATCTTCAAGCCAGTGCG CAAGCACTTTGATATGCCTCATGATATTGTTGCATCTGAAGATGGGACTGTGTACATTGGAGATGCTCATACCAACACCGTGTGGAAGTTCACCTTGACT GAGAAATTGGAACATCGATCAGTTAAAAAGGCTGGCATTGAGGTCCAGGAAATCAAAGGAAAGGGAAGTGGAGGCTTAAACCTTGGTAATTTCTTTGCAA GCCGTAAGGGCTACAGTCGAAAAGGGTTTGACCGGCTTAGCACTGAGGGCAGTGACCAAGAGAAAGAGGATGATGGAAGTGAATCAGAAGAGGAGTATTC AGCACCTCTGCCTGCGCTCGCACCTTCCTCCTCCTGAAAACCAAGCTTTGATTTAGATTGAGTAAGATTTACCCAGAATGTCAGATTCCTTTCCCTTTAG CACGTTTAAAGTTCTGTGTATTTAATTGTAAACTGTACTAGTCTGTGTGGGACTGTACACACTTTATTTACTTCGTTTTGGTTAAGTTGGCTTCTGTTTC TAGTTGAGGAGTTTCCTAAAAGTTCATAACAGTGCCATTGTCTTTATATGAACATAGACTAGAGAAACCGTCCTCTTTTTCCATCATAATTCTAATCTAA CAATGGAAGATTTGCCCATTTACACTTTTGAGACTTTTTGGTGGATGTAAATAACCCCATTCTTTGCTTGAACACAGTATTTTCCCAATAGCACTTTCAT TGCCAGTGTCTTTCTTTGGTGCCTTTCCTGTTCAGCATTCTTAGCCTGTGGCAGTAAAGAGAAACTTTGTGCTACATGACGACAAAGCTGCTAAATCTCC TATTTTTTTAAAATCACTAACATTATATTGCAATGAAGGAAATAAAAAAGTCTCTATTTAAATTCTTTTTTAAATTTTCTTCAGTTGGTGTGTTTTTGGG ATGTCTTATTTTTAGATGGTTACACTGTTAGAACACTATTTTCAGAATCTGAATGTAATTTGTGTAATAAAGTGTTTTCAGAGCA

refseq_PAM.F1 (forward primer)
refseq_PAM.R1 (reverse primer)
Single exon skipping

Frame 0

VLNDD*RGFG*YPSQPLSFRSHKAPARLAPAPG HASGAGGRKASACGPDKSPACPRLFARRS*PRRL AAASSLLSLADGVWPPQDARRARAMK*RLLAAP LPNRQPQPRAALPGPLPAGSYRRGHGWPRP*PA SSPCFSKQLFGFPKPTFCL*EV*RNYQTIFQ*MS WYHQTRSSY*FIRFCIGYSHAWGYT*TVRYILL HVYANTSG*GSLRD*LQASSQHGYCPSHVTFWM QYAFIHWKLLVL**RNLYR*SQYSVCLGEKCSPY PAPQRCWIQSWRRDWK*ILCTTGTLWGY*CF*R **QGLFWCVLTPHTSATAFNCWHVPYDVC*HCY PSRRKSGEF*HFMPL*KLSNACLCL*SSHSPFR* GSKWIQSKKWTVDTDWTAEPSAATGFLPCGASS *CKFW*PTGCKMCIHW*RKDRSHTHWWHV***N VQLIHYVLHGSQACSFFHDLYPECSSRYVQNHTT RGQHSNSREV*YGYDA*TS*RNRI*R*DSFTTA AKTRRRRSVRPG*FLFTTFQAARRKGRCCSCAQ I*SYRKGRIRVRPGS*DCKCSPKKGSWSI*CQRG CRT*EG*CYSCQRQNSQIPQTSIYLEATREQSF LITAAPTW*RHLGTRTHRRFPHGRGTGLAWSIL VTRPGFWGGSRP*E*PGDFPQR*PCLGWKLV*QQ VCLPANRTRTN*RRHYSCHRSK*CCSTPVQWKK SVLLATWLEYR*RWELLGHRRGSPSGVQTGSKQ *RRPCINPGKEHATRQ*PESLLSTH*CGCGSRHW SHLCIRWLLQQQDCAVFTKWKVHHTVGRRVFRE QSSARPVHCSSQLGSCASFGPIMCGRPGKWSDP VF*N*HQRICERD*AFIIWKKCICNFIYTRLALC SEWEASFWGPRTCTRICDELFQWGNYRHLQASA QAL*YAS*YCCI*RWDCVHWRCSYQHRVEVHLD *EIGTSIS*KGWH*GPGNQRKGKWRLKPW*FLCK P*GLQSKRV*PA*H*GQ*PRERG*WK*IRRGVF STSACARTFLLLKTKL*FRLSKIYPECQIPFPL ARLKFCVFNCKLY*SVWDCTHFIYFVLVKLASVS S*GVS*KFITVPLSLYEHRLEKPSSFSIIILI* QWKICPFTLLRLFGGCK*PHSLLEHSIFPIALS LPVSFFGAFPVQHS*PVAVKRNFVLHDDKAAKSP IFLKSLTLYCNEGNKKVSI*ILF*IFFSWCVFG MSYF*MVTLLEHYFQNLNVICVIKCFQS

Frame 1

F*MMTDAGLGDTPHSPCHSGVIRHPRV*PQRQG TRAALEGGKLPPAGRTKVPPAHGFLPAARDRDA SPRPARCSRWRMVCGRRRTPAVPGP*SSGCWRRR CPTASPSPALRCPVLSRRGRIGVDMAGRVPSLL VLLVFPSSCLAFRSPLSVFKRFKETTRPFSNEC LGTTRPVVPIDSSDFALDIRMPGVTPKQSDTYFC MSMRIPVDEEAFVIDFKPRASMDTVHHMLLFGC NMPSSTGSYWFCDEGTCTDKANILYAWARNAPP TRLPKGVGFRVGGETGSKYFVLQVHYGDISAFRD NNKDCSGVSLHLTRLPQPLIAGMYLMMSVDTVI PAGEKVVNSDISCHYKNYPMHVFAYRVHTHHLG KVVSGYRVRNGQWTLIGRQSPQLPQAFYPVGHPV DVSFGDLLAARCVFTGEGRTEATHIGGTSSDEM CNLYIMYYMEAKHAVSFMTCTQNVAPDMFRTIP PEANIPIPVKSDMVMMHEHHKETEYKDKIPLLQQ PKREEEEVLDQGDFYSLLSKLLGEREDVVHVHK YNPTEKAESESDLVAEIANVVQKKDLGRSDARE GAEHERGNAILVRDRIHKFHRLVSTLRPPESRVF SLQQPPPGEGTWEPEHTGDFHMEEALDWPGVYL LPGQVSGVALDPKNNLVIFHRGDHVWDGNSFDS KFVYQQIGLGPIEEDTILVIDPNNAAVLQSSGKN LFYLPHGLSIDKDGNYWVTDVALHQVFKLDPNN KEGPVLILGRSMQPGSDQNHFCQPTDVAVDPGT GAIYVSDGYCNSRIVQFSPSGKFITQWGEESSGS SPLPGQFTVPHSLALVPLLGQLCVADRENGRIQ CFKTDTKEFVREIKHSSFGRNVFAISYIPGLLF AVNGKPHFGDQEPVQGFVMNFSNGEIIDIFKPVR KHFDMPHDIVASEDGTVYIGDAHTNTVWKFTLT EKLEHRSVKKAGIEVQEIKGKGSGGLNLGNFFA SRKGYSRKGFDRLSTEGSDQEKEDDGSESEEEYS APLPALAPSSS*KPSFDLD*VRFTQNVRFLSL* HV*SSVYLIVNCTSLCGTVHTLFTSFWLSWLLF LVEEFPKSS*QCHCLYMNID*RNRPLFPS*F*SN NGRFAHLHF*DFLVDVNNPILCLNTVFSQ*HFH CQCLSLVPFLFSILSLWQ*RETLCYMTTKLLNL LFF*NH*HYIAMKEIKKSLFKFFFKFSSVGVFLG CLIFRWLHC*NTIFRI*M*FV**SVFRA

Frame 2

SE**LTRVWVIPLTAPVIPES*GTRASSPSARA RERRWREESFRLRAGQKSRLPTAFCPPLVTETP RRGQLAALAGGWCVAAAGRPPCPGHEVAAAGGA AAQPPAPAPRCAARSSPGGVVSAWTWLAASLAC* FSLFFQAAVWLSEAHFLSLRGLKKLPDHFPMNV LVPPDP*FLLIHQILHWIFACLGLHLNSPIHTS ACLCEYQWMRKPS*LTSSLEPAWILSITCYFLDA ICLHPLEVTGFVMKEPVQIKPIFCMPGREMLPL PGSPKVLDSELEERLEVNTLYYRYTMGILVLLE IITRTVLVCPYTSHVCHSL*LLACTL*CLLTLLS QQEKKW*ILTFHAIIKIIQCMSLPIEFTLTI*V R**VDTE*EMDSGH*LDGRALSCHRLSTLWGIQ LM*VLVTYWLQDVYSLVKEGQKPHTLVARLVMKC ATYTLCITWKPSMQFLS*PVPRM*LQICSEPYH QRPTFQFP*SLIWL*CMNIIKKQNIKIRFLYYS SQNEKKKKC*TRVISIHYFPSC*EKGKMLFMCTN IILQKRQNQSQTW*LRLQM*SKKRILVDLMPER VQNMRGVMLFLSETEFTNSTD*YLP*GHQRAEF SHYSSPHLVKAPGNQNTQEISTWKRHWIGLEYTC YQARFLGWL*TLRITW*FSTEVTMSGMETRLTA SLFTSK*DSDQLKKTLFLS*IQIMLQYSSPVEK ICFTCHMA*V*IKMGIIGSQTWLSIRCSNWIQTI KKALY*SWEGACNQAVTRITSVNPLMWLWIQAL EPFMYQMVTATAGLCSFHQVESSSHSGEKSLQG AVLCQASSLFLTAWLLCLFWANYVWQTGKMVGSS VLKLTPKNL*ERLSIHHLEEMYLQFHIYQACSL Q*MGSLILGTKNLYKDL**TFPMGKL*TSSSQC ASTLICLMILLHLKMGLCTLEMLIPTPCGSSP*L RNWNIDQLKRLALRSRKSKEREVEA*TLVISLQ AVRATVEKGLTGLALRAVTKRKRMMEVNQKRSI QHLCLRSHLPPPENQALI*IE*DLPRMSDSFPFS TFKVLCI*L*TVLVCVGLYTLYLLRFG*VGFCF *LRSFLKVHNSAIVFI*T*TRETVLFFHHNSNL TMEDLPIYTFETFWWM*ITPFFA*TQYFPNSTFI ASVFLWCLSCSAFLACGSKEKLCAT*RQSC*IS YFFKITNIILQ*RK*KSLYLNSFLNFLQLVCFW DVLFLDGYTVRTLFSESECNLCNKVFSE