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Sequences

Sequence PCSK6.kAug05

PCSK6.F1 (forward primer)
PCSK6.R33 (reverse primer)
Single exon skipping

Frame 0

Frame 1

Frame 2

Sequence PCSK6.iAug05

PCSK6.F1 (forward primer)
PCSK6.R33 (reverse primer)
Single exon skipping

Frame 0

Frame 1

Frame 2

Sequence PCSK6.hAug05

TCGCGGGCCGAGGACGCCTCTGGGGCGGCACCGCGTCCCGAGAGCCCCAGAAGTCGGCGGGGAAGTTCCCCCGGTGGGGGGCGTTTCGGGCCTCCCGGAC GGCTCTCGGCCCCGGAGCCCGGTCGCAGGAGCGCGGGCCCGGGGGCGGGAACGCGCCGCGGCCGCCTCCTCCTCCCCGGCTCCCGCCCGCGGCGGTGTTG GCGGCGGCGGTGGCGGCGGCGGCGGCGCTTCCCCGGCGCGGAGCGGCTTTAAAAGGCGGCACTCCACCCCCCGGCGCACTCGCAGCTCGGGCGCCGCGCG AGCCTGTCGCCGCTATGCCTCCGCGCGCGCCGCCTGCGCCCGGGCCCCGGCCGCCGCCCCGGGCCGCCGCCGCCACCGACACCGCCGCGGGCGCGGGGGG CGCGGGGGGCGCGGGGGGCGCCGGCGGGCCCGGGTTCCGGCCGCTCGCGCCGCGTCCCTGGCGCTGGCTGCTGCTGCTGGCGCTGCCTGCCGCCTGCTCC GCCCCCGCCGCGCCCCGTCTACACCAACCACTGGGCGGTGCAAGTGCTGGGCGGCCCGGCCGAGGCGGACCGCGTGGCGGCGGCGCACGGGTACCTCAAC TTGGGCCAGATTGGAAACCTGGAAGATTACTACCATTTTTATCACAGCAAAACCTTTAAAAGATCAACCTTGAGTAGCAGAGGCCCTCACACCTTCCTCA GAATGGACCCCCAGGTGAAATGGCTCCAGCAACAGGAAGTGAAACGAAGGGTGAAGAGACAGGTGCGAAGTGACCCGCAGGCCCTTTACTTCAACGACCC CATTTGGTCCAACATGTGGTACCTGCATTGTGGCGACAAGAACAGTCGCTGCCGGTCGGAAATGAATGTCCAGGCAGCGTGGAAGAGGGGCTACACAGGA AAAAACGTGGTGGTCACCATCCTTGATGATGGCATAGAGAGAAATCACCCTGACCTGGCCCCAAATTATGATTCCTACGCCAGCTACGACGTGAACGGCA ATGATTATGACCCATCTCCACGATATGATGCCAGCAATGAAAATAAACACGGCACTCGTTGTGCGGGAGAAGTTGCTGCTTCAGCAAACAATTCCTACTG CATCGTGGGCATAGCGTACAATGCCAAAATAGGAGGCATCCGCATGCTGGACGGCGATGTCACAGATGTGGTCGAGGCAAAGTCGCTGGGCATCAGACCC AACTACATCGACATTTACAGTGCCAGCTGGGGGCCGGACGACGACGGCAAGACGGTGGACGGGCCCGGCCGACTGGCTAAGCAGGCTTTCGAGTATGGCA TTAAAAAGGGCCGGCAGGGCCTGGGCTCCATTTTCGTCTGGGCATCTGGGAATGGCGGGAGAGAGGGGGACTACTGCTCGTGCGATGGCTACACCAACAG CATCTACACCATCTCCGTCAGCAGCGCCACCGAGAATGGCTACAAGCCCTGGTACCTGGAAGAGTGTGCCTCCACCCTGGCCACCACCTACAGCAGTGGG GCCTTTTATGAGCGAAAAATCGTCACCACGGATCTGCGTCAGCGCTGTACCGATGGCCACACTGGGACCTCAGTCTCTGCCCCCATGGTGGCGGGCATCA TCGCCTTGGCTCTAGAAGCAAACAGCCAGTTAACCTGGAGGGACGTCCAGCACCTGCTAGTGAAGACATCCCGGCCGGCCCACCTGAAAGCGAGCGACTG GAAAGTGAACGGCGCGGGTCATAAAGTTAGCCATTTCTATGGATTTGGTTTGGTGGACGCAGAAGCTCTCGTTGTGGAGGCAAAGAAGTGGACAGCAGTG CCATCGCAGCACATGTGTGTGGCCGCCTCGGACAAGAGACCCAGGAGCATCCCCTTAGTGCAGGTGCTGCGGACTACGGCCCTGACCAGCGCCTGCGCGG AGCACTCGGACCAGCGGGTGGTCTACTTGGAGCACGTGGTGGTTCGCACCTCCATCTCACACCCACGCCGAGGAGACCTCCAGATCTACCTGGTTTCTCC CTCGGGAACCAAGTCTCAACTTCTGGCAAAGAGGTTGCTGGATCTTTCCAATGAAGGGTTTACAAACTGGGAATTCATGACTGTCCACTGCTGGGGAGAA AAGGCTGAAGGGCAGTGGACCTTGGAAATCCAAGATCTGCCATCCCAGGTCCGCAACCCGGAGAAGCAAGGGAACCTGGACTAAAACACGTGTTCCGGTG GCAGATTGAACAAGAGCTTTGGTAATCTCTGTGAAATACTGCAGAGGGACAGATGACCAAAGCCACCACATTTAGAACTTTGGCTGCCTTTGGAAGTCCA GAGCTGGATCTCTCAGCTCCCGCCCCCAGAGGCTCAGCACTTTGGACATGGCTCACAAACAGTTGTTGATTGACTGCATGAATGCGTGTGCGTGCAAGCA TGAACCTTGTTTAAATCAAGAGCTTACATAATTTTAACCAGTTCTGTCTTCAGCTGTACATACTCAGTAAAATGTTTAATGAAGGGGAAGAGATTAGTCT CTTCTGTGTGACCATGTTTTCCCTTTATTCATCCTAAAAAGTTCCATGAATTCTTGATTTCCTTTCAGTGGCCCTTTCAACAATGTCTTTTTTCCCAAGA GCATAACTGTTCTCATTTTATTGCTAGCCATCTTGATCTGTGTTTTATTGACATCTCTTTTGAGCTAATCTTCATTTCTAAGATAAGAGTTGAGATTTTG CAATCTGTGTTCGATGGCTCAATCTATCCTGTGCTTGATGCTAGAAAGGAAGACAGATTTAAAGCACATGCCTTCTGTGCCGGCTTTCAAGTTTGTCACT AAACTCTCATTTCTGGAAAGTGCAATTATAGAGTATCACTCCCACTTCCTTGGAAACAGAGCTGAAGAACTTGGCACACTCTCCAAACAGTCACCATACA CACTGTTGTCAAAAAGTTCCATTTTTAACCCCATTTGCATTAATATTGCAGTCAATCTCTTTACCTCCTTTTCTCTTTCACGGGGCCGTGACAGTGACGC CTTTCCCCAAAACTCTCCTCGTTTGAGAAAAAAAGAAGTATGTATCCCCACTTATCTCGGGGAGAAATGCAACCAACTGCTGCTGTGCACATTTATGAAT CACAGTATTGTTTAGTCGGTTCTGTATCTCCAGTAGAAAGCATAACAAAAAGATGACCTTTGTCTCACCTCATAGCTAATTTTTGCAAATAAAATACTAA ACATTGATGAAAATGAAAATGACAGATCTGAGGAAGAGGGAGAGCTCTCTTGAAACCCCTTTCCCAGATTTGCCCTCAGTTTTAGGAATGAGGTACGCTT GGGCATTCCTGTTGCCTGCATGGTTCGGCATTCTGGCAGCCAGGTTCGCCCCAGAATAGATAAGTGTGTTTGATTTTCGAACATTTGACTTTATTACCTA TTTCCAGAAACACTTCGTGTTCAAACTACGTCTCTACAGGCATTTCTACTTGGTCTTTTTCTCCCCTGACATTTCACAGAGTCCTAACTGTGCTGCATGT ATAACTTGTTCTTTCTTTTTGTTACTTGCATAGCAAGCATGTTTCTATGTCATTAAATATCCTTTGTAAGCAGC

PCSK6.F1 (forward primer)
PCSK6.R33 (reverse primer)
Single exon skipping

Frame 0

SRAEDASGAAPRPESPRSRRGSSPGGGRFGPPG RLSAPEPGRRSAGPGAGTRRGRLLLPGSRPRRCW RRRWRRRRRFPGAERL*KAALHPPAHSQLGRRA SLSPLCLRARRLRPGPGRRPGPPPPPTPPRARG ARGARGAPAGPGSGRSRRVPGAGCCCWRCLPPAP PPPRPVYTNHWAVQVLGGPAEADRVAAAHGYLN LGQIGNLEDYYHFYHSKTFKRSTLSSRGPHTFL RMDPQVKWLQQQEVKRRVKRQVRSDPQALYFNDP IWSNMWYLHCGDKNSRCRSEMNVQAAWKRGYTG KNVVVTILDDGIERNHPDLAPNYDSYASYDVNG NDYDPSPRYDASNENKHGTRCAGEVAASANNSYC IVGIAYNAKIGGIRMLDGDVTDVVEAKSLGIRP NYIDIYSASWGPDDDGKTVDGPGRLAKQAFEYG IKKGRQGLGSIFVWASGNGGREGDYCSCDGYTNS IYTISVSSATENGYKPWYLEECASTLATTYSSG AFYERKIVTTDLRQRCTDGHTGTSVSAPMVAGI IALALEANSQLTWRDVQHLLVKTSRPAHLKASDW KVNGAGHKVSHFYGFGLVDAEALVVEAKKWTAV PSQHMCVAASDKRPRSIPLVQVLRTTALTSACA EHSDQRVVYLEHVVVRTSISHPRRGDLQIYLVSP SGTKSQLLAKRLLDLSNEGFTNWEFMTVHCWGE KAEGQWTLEIQDLPSQVRNPEKQGNLD*NTCSG GRLNKSFGNLCEILQRDR*PKPPHLELWLPLEVQ SWISQLPPPEAQHFGHGSQTVVD*LHECVCVQA *TLFKSRAYIILTSSVFSCTYSVKCLMKGKRLV SSV*PCFPFIHPKKFHEFLISFQWPFQQCLFSQE HNCSHFIASHLDLCFIDISFELIFISKIRVEIL QSVFDGSIYPVLDARKEDRFKAHAFCAGFQVCH *TLISGKCNYRVSLPLPWKQS*RTWHTLQTVTIH TVVKKFHF*PHLH*YCSQSLYLLFSFTGP*Q*R LSPKLSSFEKKRSMYPHLSRGEMQPTAAVHIYE SQYCLVGSVSPVESITKR*PLSHLIANFCK*NTK H**K*K*QI*GRGRALLKPLSQICPQF*E*GTL GHSCCLHGSAFWQPGSPQNR*VCLIFEHLTLLP ISRNTSCSNYVSTGISTWSFSPLTFHRVLTVLHV *LVLSFCYLHSKHVSMSLNILCKQ

Frame 1

RGPRTPLGRHRVPRAPEVGGEVPPVGGVSGLPD GSRPRSPVAGARARGRERAAAASSSPAPARGGV GGGGGGGGGASPARSGFKRRHSTPRRTRSSGAAR ACRRYASARAACARAPAAAPGRRRHRHRRGRGG RGGRGGRRRARVPAARAASLALAAAAGAACRLL RPRRAPSTPTTGRCKCWAARPRRTAWRRRTGTST WARLETWKITTIFITAKPLKDQP*VAEALTPSS EWTPR*NGSSNRK*NEG*RDRCEVTRRPFTSTT PFGPTCGTCIVATRTVAAGRK*MSRQRGRGATQE KTWWSPSLMMA*REITLTWPQIMIPTPATT*TA MIMTHLHDMMPAMKINTALVVREKLLLQQTIPT ASWA*RTMPK*EASACWTAMSQMWSRQSRWASDP TTSTFTVPAGGRTTTARRWTGPADWLSRLSSMA LKRAGRAWAPFSSGHLGMAGERGTTARAMATPT ASTPSPSAAPPRMATSPGTWKSVPPPWPPPTAVG PFMSEKSSPRICVSAVPMATLGPQSLPPWWRAS SPWL*KQTAS*PGGTSSTC**RHPGRPT*KRAT GK*TARVIKLAISMDLVWWTQKLSLWRQRSGQQC HRSTCVWPPRTRDPGASP*CRCCGLRP*PAPAR STRTSGWSTWSTWWFAPPSHTHAEETSRSTWFL PREPSLNFWQRGCWIFPMKGLQTGNS*LSTAGEK RLKGSGPWKSKICHPRSATRRSKGTWTKTRVPV AD*TRALVISVKYCRGTDDQSHHI*NFGCLWKS RAGSLSSRPQRLSTLDMAHKQLLIDCMNACACKH EPCLNQELT*F*PVLSSAVHTQ*NV**RGRD*S LLCDHVFPLFILKSSMNS*FPFSGPFNNVFFPK SITVLILLLAILICVLLTSLLS*SSFLR*ELRFC NLCSMAQSILCLMLERKTDLKHMPSVPAFKFVT KLSFLESAIIEYHSHFLGNRAEELGTLSKQSPY TLLSKSSIFNPICINIAVNLFTSFSLSRGRDSDA FPQNSPRLRKKEVCIPTYLGEKCNQLLLCTFMN HSIV*SVLYLQ*KA*QKDDLCLTS*LIFANKIL NIDENENDRSEEEGELS*NPFPRFALSFRNEVRL GIPVACMVRHSGSQVRPRIDKCV*FSNI*LYYL FPETLRVQTTSLQAFLLGLFLP*HFTES*LCCM YNLFFLFVTCIASMFLCH*ISFVSS

Frame 2

AGRGRLWGGTASREPQKSAGKFPRWGAFRASRT ALGPGARSQERGPGGGNAPRPPPPPRLPPAAVL AAAVAAAAALPRRGAALKGGTPPPGALAARAPR EPVAAMPPRAPPAPGPRPPPRAAAATDTAAGAGG AGGAGGAGGPGFRPLAPRPWRWLLLLALPAACS APAAPRLHQPLGGASAGRPGRGGPRGGGARVPQ LGPDWKPGRLLPFLSQQNL*KINLE*QRPSHLPQ NGPPGEMAPATGSETKGEETGAK*PAGPLLQRP HLVQHVVPALWRQEQSLPVGNECPGSVEEGLHR KKRGGHHP**WHREKSP*PGPKL*FLRQLRRERQ *L*PISTI*CQQ*K*TRHSLCGRSCCFSKQFLL HRGHSVQCQNRRHPHAGRRCHRCGRGKVAGHQT QLHRHLQCQLGAGRRRQDGGRARPTG*AGFRVWH *KGPAGPGLHFRLGIWEWRERGGLLLVRWLHQQ HLHHLRQQRHREWLQALVPGRVCLHPGHHLQQW GLL*AKNRHHGSASALYRWPHWDLSLCPHGGGHH RLGSRSKQPVNLEGRPAPASEDIPAGPPESERL ESERRGS*S*PFLWIWFGGRRSSRCGGKEVDSS AIAAHVCGRLGQETQEHPLSAGAADYGPDQRLRG ALGPAGGLLGARGGSHLHLTPTPRRPPDLPGFS LGNQVSTSGKEVAGSFQ*RVYKLGIHDCPLLGR KG*RAVDLGNPRSAIPGPQPGEAREPGLKHVFRW QIEQELW*SL*NTAEGQMTKATTFRTLAAFGSP ELDLSAPAPRGSALWTWLTNSC*LTA*MRVRAS MNLV*IKSLHNFNQFCLQLYILSKMFNEGEEISL FCVTMFSLYSS*KVP*ILDFLSVALSTMSFFPR A*LFSFYC*PS*SVFY*HLF*ANLHF*DKS*DF AICVRWLNLSCA*C*KGRQI*STCLLCRLSSLSL NSHFWKVQL*SITPTSLETELKNLAHSPNSHHT HCCQKVPFLTPFALILQSISLPPFLFHGAVTVT PFPKTLLV*EKKKYVSPLISGRNATNCCCAHL*I TVLFSRFCISSRKHNKKMTFVSPHS*FLQIKY* TLMKMKMTDLRKRESSLETPFPDLPSVLGMRYA WAFLLPAWFGILAARFAPE*ISVFDFRTFDFITY FQKHFVFKLRLYRHFYLVFFSPDISQSPNCAAC ITCSFFLLLA*QACFYVIKYPL*A

Sequence PCSK6.gAug05

TCGCGGGCCGAGGACGCCTCTGGGGCGGCACCGCGTCCCGAGAGCCCCAGAAGTCGGCGGGGAAGTTCCCCCGGTGGGGGGCGTTTCGGGCCTCCCGGAC GGCTCTCGGCCCCGGAGCCCGGTCGCAGGAGCGCGGGCCCGGGGGCGGGAACGCGCCGCGGCCGCCTCCTCCTCCCCGGCTCCCGCCCGCGGCGGTGTTG GCGGCGGCGGTGGCGGCGGCGGCGGCGCTTCCCCGGCGCGGAGCGGCTTTAAAAGGCGGCACTCCACCCCCCGGCGCACTCGCAGCTCGGGCGCCGCGCG AGCCTGTCGCCGCTATGCCTCCGCGCGCGCCGCCTGCGCCCGGGCCCCGGCCGCCGCCCCGGGCCGCCGCCGCCACCGACACCGCCGCGGGCGCGGGGGG CGCGGGGGGCGCGGGGGGCGCCGGCGGGCCCGGGTTCCGGCCGCTCGCGCCGCGTCCCTGGCGCTGGCTGCTGCTGCTGGCGCTGCCTGCCGCCTGCTCC GCCCCCGCCGCGCCCCGTCTACACCAACCACTGGGCGGTGCAAGTGCTGGGCGGCCCGGCCGAGGCGGACCGCGTGGCGGCGGCGCACGGGTACCTCAAC TTGGGCCAGATTGGAAACCTGGAAGATTACTACCATTTTTATCACAGCAAAACCTTTAAAAGATCAACCTTGAGTAGCAGAGGCCCTCACACCTTCCTCA GAATGGACCCCCAGGTGAAATGGCTCCAGCAACAGGAAGTGAAACGAAGGGTGAAGAGACAGGTGCGAAGTGACCCGCAGGCCCTTTACTTCAACGACCC CATTTGGTCCAACATGTGGTACCTGCATTGTGGCGACAAGAACAGTCGCTGCCGGTCGGAAATGAATGTCCAGGCAGCGTGGAAGAGGGGCTACACAGGA AAAAACGTGGTGGTCACCATCCTTGATGATGGCATAGAGAGAAATCACCCTGACCTGGCCCCAAATTATGATTCCTACGCCAGCTACGACGTGAACGGCA ATGATTATGACCCATCTCCACGATATGATGCCAGCAATGAAAATAAACACGGCACTCGTTGTGCGGGAGAAGTTGCTGCTTCAGCAAACAATTCCTACTG CATCGTGGGCATAGCGTACAATGCCAAAATAGGAGGCATCCGCATGCTGGACGGCGATGTCACAGATGTGGTCGAGGCAAAGTCGCTGGGCATCAGACCC AACTACATCGACATTTACAGTGCCAGCTGGGGGCCGGACGACGACGGCAAGACGGTGGACGGGCCCGGCCGACTGGCTAAGCAGGCTTTCGAGTATGGCA TTAAAAAGGGCCGGCAGGGCCTGGGCTCCATTTTCGTCTGGGCATCTGGGAATGGCGGGAGAGAGGGGGACTACTGCTCGTGCGATGGCTACACCAACAG CATCTACACCATCTCCGTCAGCAGCGCCACCGAGAATGGCTACAAGCCCTGGTACCTGGAAGAGTGTGCCTCCACCCTGGCCACCACCTACAGCAGTGGG GCCTTTTATGAGCGAAAAATCGTCACCACGGATCTGCGTCAGCGCTGTACCGATGGCCACACTGGGACCTCAGTCTCTGCCCCCATGGTGGCGGGCATCA TCGCCTTGGCTCTAGAAGCAAACAGCCAGTTAACCTGGAGGGACGTCCAGCACCTGCTAGTGAAGACATCCCGGCCGGCCCACCTGAAAGCGAGCGACTG GAAAGTGAACGGCGCGGGTCATAAAGTTAGCCATTTCTATGGATTTGGTTTGGTGGACGCAGAAGCTCTCGTTGTGGAGGCAAAGAAGTGGACAGCAGTG CCATCGCAGCACATGTGTGTGGCCGCCTCGGACAAGAGACCCAGGAGCATCCCCTTAGTGCAGGTGCTGCGGACTACGGCCCTGACCAGCGCCTGCGCGG AGCACTCGGACCAGCGGGTGGTCTACTTGGAGCACGTGGTGGTTCGCACCTCCATCTCACACCCACGCCGAGGAGACCTCCAGATCTACCTGGTTTCTCC CTCGGGAACCAAGTCTCAACTTCTGGCAAAGAGGTTGCTGGATCTTTCCAATGAAGGGTTTACAAACTGGGAATTCATGACTGTCCACTGCTGGGGAGAA AAGGCTGAAGGGCAGTGGACCTTGGAAATCCAAGATCTGCCATCCCAGGTCCGCAACCCGGAGAAGCAAGGTGATCTTGAGACTCCTGTTGCAAATCAAC TGACCACAGAAGAGAGGGAACCTGGACTAAAACACGTGTTCCGGTGGCAGATTGAACAAGAGCTTTGGTAATCTCTGTGAAATACTGCAGAGGGACAGAT GACCAAAGCCACCACATTTAGAACTTTGGCTGCCTTTGGAAGTCCAGAGCTGGATCTCTCAGCTCCCGCCCCCAGAGGCTCAGCACTTTGGACATGGCTC ACAAACAGTTGTTGATTGACTGCATGAATGCGTGTGCGTGCAAGCATGAACCTTGTTTAAATCAAGAGCTTACATAATTTTAACCAGTTCTGTCTTCAGC TGTACATACTCAGTAAAATGTTTAATGAAGGGGAAGAGATTAGTCTCTTCTGTGTGACCATGTTTTCCCTTTATTCATCCTAAAAAGTTCCATGAATTCT TGATTTCCTTTCAGTGGCCCTTTCAACAATGTCTTTTTTCCCAAGAGCATAACTGTTCTCATTTTATTGCTAGCCATCTTGATCTGTGTTTTATTGACAT CTCTTTTGAGCTAATCTTCATTTCTAAGATAAGAGTTGAGATTTTGCAATCTGTGTTCGATGGCTCAATCTATCCTGTGCTTGATGCTAGAAAGGAAGAC AGATTTAAAGCACATGCCTTCTGTGCCGGCTTTCAAGTTTGTCACTAAACTCTCATTTCTGGAAAGTGCAATTATAGAGTATCACTCCCACTTCCTTGGA AACAGAGCTGAAGAACTTGGCACACTCTCCAAACAGTCACCATACACACTGTTGTCAAAAAGTTCCATTTTTAACCCCATTTGCATTAATATTGCAGTCA ATCTCTTTACCTCCTTTTCTCTTTCACGGGGCCGTGACAGTGACGCCTTTCCCCAAAACTCTCCTCGTTTGAGAAAAAAAGAAGTATGTATCCCCACTTA TCTCGGGGAGAAATGCAACCAACTGCTGCTGTGCACATTTATGAATCACAGTATTGTTTAGTCGGTTCTGTATCTCCAGTAGAAAGCATAACAAAAAGAT GACCTTTGTCTCACCTCATAGCTAATTTTTGCAAATAAAATACTAAACATTGATGAAAATGAAAATGACAGATCTGAGGAAGAGGGAGAGCTCTCTTGAA ACCCCTTTCCCAGATTTGCCCTCAGTTTTAGGAATGAGGTACGCTTGGGCATTCCTGTTGCCTGCATGGTTCGGCATTCTGGCAGCCAGGTTCGCCCCAG AATAGATAAGTGTGTTTGATTTTCGAACATTTGACTTTATTACCTATTTCCAGAAACACTTCGTGTTCAAACTACGTCTCTACAGGCATTTCTACTTGGT CTTTTTCTCCCCTGACATTTCACAGAGTCCTAACTGTGCTGCATGTATAACTTGTTCTTTCTTTTTGTTACTTGCATAGCAAGCATGTTTCTATGTCATT AAATATCCTTTGTAAGCAGC

PCSK6.F1 (forward primer)
PCSK6.R33 (reverse primer)
Single exon skipping

Frame 0

SRAEDASGAAPRPESPRSRRGSSPGGGRFGPPG RLSAPEPGRRSAGPGAGTRRGRLLLPGSRPRRCW RRRWRRRRRFPGAERL*KAALHPPAHSQLGRRA SLSPLCLRARRLRPGPGRRPGPPPPPTPPRARG ARGARGAPAGPGSGRSRRVPGAGCCCWRCLPPAP PPPRPVYTNHWAVQVLGGPAEADRVAAAHGYLN LGQIGNLEDYYHFYHSKTFKRSTLSSRGPHTFL RMDPQVKWLQQQEVKRRVKRQVRSDPQALYFNDP IWSNMWYLHCGDKNSRCRSEMNVQAAWKRGYTG KNVVVTILDDGIERNHPDLAPNYDSYASYDVNG NDYDPSPRYDASNENKHGTRCAGEVAASANNSYC IVGIAYNAKIGGIRMLDGDVTDVVEAKSLGIRP NYIDIYSASWGPDDDGKTVDGPGRLAKQAFEYG IKKGRQGLGSIFVWASGNGGREGDYCSCDGYTNS IYTISVSSATENGYKPWYLEECASTLATTYSSG AFYERKIVTTDLRQRCTDGHTGTSVSAPMVAGI IALALEANSQLTWRDVQHLLVKTSRPAHLKASDW KVNGAGHKVSHFYGFGLVDAEALVVEAKKWTAV PSQHMCVAASDKRPRSIPLVQVLRTTALTSACA EHSDQRVVYLEHVVVRTSISHPRRGDLQIYLVSP SGTKSQLLAKRLLDLSNEGFTNWEFMTVHCWGE KAEGQWTLEIQDLPSQVRNPEKQGDLETPVANQ LTTEEREPGLKHVFRWQIEQELW*SL*NTAEGQM TKATTFRTLAAFGSPELDLSAPAPRGSALWTWL TNSC*LTA*MRVRASMNLV*IKSLHNFNQFCLQ LYILSKMFNEGEEISLFCVTMFSLYSS*KVP*IL DFLSVALSTMSFFPRA*LFSFYC*PS*SVFY*H LF*ANLHF*DKS*DFAICVRWLNLSCA*C*KGR QI*STCLLCRLSSLSLNSHFWKVQL*SITPTSLE TELKNLAHSPNSHHTHCCQKVPFLTPFALILQS ISLPPFLFHGAVTVTPFPKTLLV*EKKKYVSPL ISGRNATNCCCAHL*ITVLFSRFCISSRKHNKKM TFVSPHS*FLQIKY*TLMKMKMTDLRKRESSLE TPFPDLPSVLGMRYAWAFLLPAWFGILAARFAP E*ISVFDFRTFDFITYFQKHFVFKLRLYRHFYLV FFSPDISQSPNCAACITCSFFLLLA*QACFYVI KYPL*A

Frame 1

RGPRTPLGRHRVPRAPEVGGEVPPVGGVSGLPD GSRPRSPVAGARARGRERAAAASSSPAPARGGV GGGGGGGGGASPARSGFKRRHSTPRRTRSSGAAR ACRRYASARAACARAPAAAPGRRRHRHRRGRGG RGGRGGRRRARVPAARAASLALAAAAGAACRLL RPRRAPSTPTTGRCKCWAARPRRTAWRRRTGTST WARLETWKITTIFITAKPLKDQP*VAEALTPSS EWTPR*NGSSNRK*NEG*RDRCEVTRRPFTSTT PFGPTCGTCIVATRTVAAGRK*MSRQRGRGATQE KTWWSPSLMMA*REITLTWPQIMIPTPATT*TA MIMTHLHDMMPAMKINTALVVREKLLLQQTIPT ASWA*RTMPK*EASACWTAMSQMWSRQSRWASDP TTSTFTVPAGGRTTTARRWTGPADWLSRLSSMA LKRAGRAWAPFSSGHLGMAGERGTTARAMATPT ASTPSPSAAPPRMATSPGTWKSVPPPWPPPTAVG PFMSEKSSPRICVSAVPMATLGPQSLPPWWRAS SPWL*KQTAS*PGGTSSTC**RHPGRPT*KRAT GK*TARVIKLAISMDLVWWTQKLSLWRQRSGQQC HRSTCVWPPRTRDPGASP*CRCCGLRP*PAPAR STRTSGWSTWSTWWFAPPSHTHAEETSRSTWFL PREPSLNFWQRGCWIFPMKGLQTGNS*LSTAGEK RLKGSGPWKSKICHPRSATRRSKVILRLLLQIN *PQKRGNLD*NTCSGGRLNKSFGNLCEILQRDR *PKPPHLELWLPLEVQSWISQLPPPEAQHFGHGS QTVVD*LHECVCVQA*TLFKSRAYIILTSSVFS CTYSVKCLMKGKRLVSSV*PCFPFIHPKKFHEF LISFQWPFQQCLFSQEHNCSHFIASHLDLCFIDI SFELIFISKIRVEILQSVFDGSIYPVLDARKED RFKAHAFCAGFQVCH*TLISGKCNYRVSLPLPW KQS*RTWHTLQTVTIHTVVKKFHF*PHLH*YCSQ SLYLLFSFTGP*Q*RLSPKLSSFEKKRSMYPHL SRGEMQPTAAVHIYESQYCLVGSVSPVESITKR *PLSHLIANFCK*NTKH**K*K*QI*GRGRALLK PLSQICPQF*E*GTLGHSCCLHGSAFWQPGSPQ NR*VCLIFEHLTLLPISRNTSCSNYVSTGISTW SFSPLTFHRVLTVLHV*LVLSFCYLHSKHVSMSL NILCKQ

Frame 2

AGRGRLWGGTASREPQKSAGKFPRWGAFRASRT ALGPGARSQERGPGGGNAPRPPPPPRLPPAAVL AAAVAAAAALPRRGAALKGGTPPPGALAARAPR EPVAAMPPRAPPAPGPRPPPRAAAATDTAAGAGG AGGAGGAGGPGFRPLAPRPWRWLLLLALPAACS APAAPRLHQPLGGASAGRPGRGGPRGGGARVPQ LGPDWKPGRLLPFLSQQNL*KINLE*QRPSHLPQ NGPPGEMAPATGSETKGEETGAK*PAGPLLQRP HLVQHVVPALWRQEQSLPVGNECPGSVEEGLHR KKRGGHHP**WHREKSP*PGPKL*FLRQLRRERQ *L*PISTI*CQQ*K*TRHSLCGRSCCFSKQFLL HRGHSVQCQNRRHPHAGRRCHRCGRGKVAGHQT QLHRHLQCQLGAGRRRQDGGRARPTG*AGFRVWH *KGPAGPGLHFRLGIWEWRERGGLLLVRWLHQQ HLHHLRQQRHREWLQALVPGRVCLHPGHHLQQW GLL*AKNRHHGSASALYRWPHWDLSLCPHGGGHH RLGSRSKQPVNLEGRPAPASEDIPAGPPESERL ESERRGS*S*PFLWIWFGGRRSSRCGGKEVDSS AIAAHVCGRLGQETQEHPLSAGAADYGPDQRLRG ALGPAGGLLGARGGSHLHLTPTPRRPPDLPGFS LGNQVSTSGKEVAGSFQ*RVYKLGIHDCPLLGR KG*RAVDLGNPRSAIPGPQPGEAR*S*DSCCKST DHRREGTWTKTRVPVAD*TRALVISVKYCRGTD DQSHHI*NFGCLWKSRAGSLSSRPQRLSTLDMA HKQLLIDCMNACACKHEPCLNQELT*F*PVLSSA VHTQ*NV**RGRD*SLLCDHVFPLFILKSSMNS *FPFSGPFNNVFFPKSITVLILLLAILICVLLT SLLS*SSFLR*ELRFCNLCSMAQSILCLMLERKT DLKHMPSVPAFKFVTKLSFLESAIIEYHSHFLG NRAEELGTLSKQSPYTLLSKSSIFNPICINIAV NLFTSFSLSRGRDSDAFPQNSPRLRKKEVCIPTY LGEKCNQLLLCTFMNHSIV*SVLYLQ*KA*QKD DLCLTS*LIFANKILNIDENENDRSEEEGELS* NPFPRFALSFRNEVRLGIPVACMVRHSGSQVRPR IDKCV*FSNI*LYYLFPETLRVQTTSLQAFLLG LFLP*HFTES*LCCMYNLFFLFVTCIASMFLCH *ISFVSS

Sequence PCSK6.fAug05

PCSK6.F1 (forward primer)
PCSK6.R33 (reverse primer)
Single exon skipping

Frame 0

Frame 1

Frame 2

Sequence PCSK6.eAug05

ACCTGGGCCAATTGGAAACCTGGAAGATTACTACCATTTTTATCACAGCAAAACCTTTAAAAGATCAACCTTGAGTAGCAGAGGCCCTCACACCTTCCTC AGAATGGACCCCCAGGTGAAATGGCTCCAGCAACAGGAAGTGAAACGAAGGGTGAAGAGACAGGTGCGAAGTGACCCGCAGGCCCTTTACTTCAACGACC CCATTTGGTCCAACATGTGGTACCTGCATTGTGGCGACAAGAACAGTCGCTGCCGGTCGGAAATGAATGTCCAGGCAGCGTGGAAGAGGGGCTACACAGG AAAAAACGTGGTGGTCACCATCCTTGATGATGGCATAGAGAGAAATCACCCTGACCTGGCCCCAAATTATGATTCCTACGCCAGCTACGACGTGAACGGC AATGATTATGACCCATCTCCACGATATGATGCCAGCAATGAAAATAAACACGGCACTCGTTGTGCGGGAGAAGTTGCTGCTTCAGCAAACAATTCCTACT GCATCGTGGGCATAGCGTACAATGCCAAAATAGGAGGCATCCGCATGCTGGACGGCGATGTCACAGATGTGGTCGAGGCAAAGTCGCTGGGCATCAGACC CAACTACATCGACATTTACAGTGCCAGCTGGGGGCCGGACGACGACGGCAAGACGGTGGACGGGCCCGGCCGACTGGCTAAGCAGGCTTTCGAGTATGGC ATTAAAAAGGGCCGGCAGGGCCTGGGCTCCATTTTCGTCTGGGCATCTGGGAATGGCGGGAGAGAGGGGGACTACTGCTCGTGCGATGGCTACACCAACA GCATCTACACCATCTCCGTCAGCAGCGCCACCGAGAATGGCTACAAGCCCTGGTACCTGGAAGAGTGTGCCTCCACCCTGGCCACCACCTACAGCAGTGG GGCCTTTTATGAGCGAAAAATCGTCACCACGGATCTGCGTCAGCGCTGTACCGATGGCCACACTGGGACCTCAGTCTCTGCCCCCATGGTGGCGGGCATC ATCGCCTTGGCTCTAGAAGCAAAGAGCATCCCCTTAGTGCAGGTGCTGCGGACTACGGCCCTGACCAGCGCCTGCGCGGAGCACTCGGACCAGCGGGTGG TCTACTTGGAGCACGTGGTGGTTCGCACCTCCATCTCACACCCACGCCGAGGAGACCTCCAGATCTACCTGGTTTCTCCCTCGGGAACCAAGTCTCAACT TCTGGCAAAGAGGTTGCTGGATCTTTCCAATGAAGGGTTTACAAACTGGGAATTCATGACTGTCCACTGCTGGGGAGAAAAGGCTGAAGGGCAGTGGACC TTGGAAATCCAAGATCTGCCATCCCAGGTCCGCAACCCGGAGAAGCAAGGGAAGTTGAAAGAATGGAGCCTCATACTGTATGGCACAGCAGAGCACCCGT ACCACACCTTCAGTGCCCATCAGTCCCGCTCGCGGATGCTGGAGCTCTCAGCCCCAGAGCTGGAGCCACCCAAGGCTGCCCTGTCACCCTCCCAGGTGGA AGTTCCTGAAGATGAGGAAGATTACACAGCTCAATCCACCCCAGGCTCTGCTAATATTTTACAGACCAGTGTGTGCCATCCGGAGTGTGGTGACAAAGGC TGTGATGGCCCCAATGCAGACCAGTGCTTGAACTGCGTCCACTTCAGCCTGGGGAGTGTCAAGACCAGCAGGAAGTGCGTGAGTGTGTGCCCCTTGGGCT ACTTTGGGGACACAGCAGCAAGACGCTGTCGCCGGTGCCACAAGGGGTGTGAGACCTGCTCCAGCAGAGCTGCGACGCAGTGCCTGTCTTGCCGCCGCGG GTTCTATCACCACCAGGAGATGAACACCTGTGTGACCCTCTGTCCTGCAGGATTTTATGCTGATGAAAGTCAGAAAAATTGCCTTAAATGCCACCCAAGC TGTAAAAAGTGCGTGGATGAACCTGAGAAATGTACTGTCTGTAAAGAAGGATTCAGCCTTGCACGGGGCAGCTGCATTCCTGACTGTGAGCCAGGCACCT ACTTTGACTCAGAGCTGATCAGATGTGGGGAATGCCATCACACCTGCGGAACCTGCGTGGGGCCAGGCAGAGAAGAGTGCATTCACTGTGCGAAAAACTT CCACTTCCACGACTGGAAGTGTGTGCCAGCCTGTGGTGAGGGCTTCTACCCAGAAGAGATGCCGGGCTTGCCCCACAAAGTGTGTCGAAGGTGTGACGAG AACTGCTTGAGCTGTGCAGGCTCCAGCAGGAACTGTAGCAGGTGTAAGACGGGCTTCACACAGCTGGGGACCTCCTGCATCACCAACCACACGTGCAGCA ACGCTGACGAGACATTCTGCGAGATGGTGAAGTCCAACCGGCTGTGCGAACGGAAGCTCTTCATTCAGTTCTGCTGCCGCACGTGCCTCCTGGCCGGGTA AGGGTGCCTAGCTGCCCACAGAGGGCAGGCACTCCCATCCATCCATCCGTCCACCTTCCTCCAGACTGTCGGCCAGAGTCTGTTTCAGGAGCGGCGCCCT GCACCTGACAGCTTTATCTCCCCAGGAGCAGCATCTCTGAGCACCCAAGCCAGGTGGGTGGTGGCTCTTAAGGAGGTGTTCCTAAAATGGTGATATCCTC TCAAATGCTGCTTGTTGGCTCCAGTCTTCCGACAAACTAACAGGAACAAAATGAATTCTGGGAATCCACAGCTCTGGCTTTGGAGCAGCTTCTGGGACCA TAAGTTTACTGAATCTTCAAGACCAAAGCAGAAAAGAAAGGCGCTTGGCATCACACATCACTCTTCTCCCCGTGCTTTTCTGCGGCTGTGTAGTAAATCT CCCCGGCCCAGCTGGCGAACCCTGGGCCATCCTCACATGTGACAAAGGGCCAGCAGTCTACCTGCTCGTTGCCTGCCACTGAGCAGTCTGGGGACGGTTT GGTCAGACTATAAATAAGATAGGTTTGAGGGCATAAAATGTATGACCACTGGGGCCGGAGTATCTATTTCTACATAGTCAGCTACTTCTGAAACTGCAGC AGTGGCTTAGAAAGTCCAATTCCAAAGCCAGACCAGAAGATTCTATCCCCCGCAGCGCTCTCCTTTGAGCAAGCCGAGCTCTCCTTGTTACCGTGTTCTG TCTGTGTCTTCAGGAGTCTCATGGCCTGAACGACCACCTCGACCTGATGCAGAGCCTTCTGAGGAGAGGCAACAGGAGGCATTCTGTGGCCAGCCAAAAG GTACCCCGATGGCCAAGCAATTCCTCTGAACAAAATGTAAAGCCAGCCATGCATTGTTAATCATCCATCACTTCCCATTTTATGGAATTGCTTTTAAAAT ACATTTGGCCTCTGCCCTTCAGAAGACTCGTTTTTAAGGTGGAAACTCCTGTGTCTGTGTATATTACAAGCCTACATGACACAGTTGGATTTATTCTGCC AAACCTGTGTAGGCATTTTATAAGCTACATGTTCTAATTTTTACCGATGTTAATTATTTTGACAAATATTTCATATATTTTCATTGAAATGCACAGATCT GCTTGATCAATTCCCTTGAATAGGGAAGTAACATTTGCCTTAAATTTTTTCGACCTCGTCTTTCTCCATATTGTCCTGCTCCCCTGTTTGACGACAGTGC ATTTGCCTTGTCACCTGTGAGCTGGAGAGAACCCAGATGTTGTTTATTGAATCTACAACTCTGAAAGAGAAATCAATGAAGCAAGTACAATGTTAACCCT AAATTAATAAAAGAGTTAACATCCCATGGCAC

PCSK6.F1 (forward primer)
PCSK6.R33 (reverse primer)
Single exon skipping

Frame 0

TWANWKPGRLLPFLSQQNL*KINLE*QRPSHLP QNGPPGEMAPATGSETKGEETGAK*PAGPLLQRP HLVQHVVPALWRQEQSLPVGNECPGSVEEGLHR KKRGGHHP**WHREKSP*PGPKL*FLRQLRRER Q*L*PISTI*CQQ*K*TRHSLCGRSCCFSKQFLL HRGHSVQCQNRRHPHAGRRCHRCGRGKVAGHQT QLHRHLQCQLGAGRRRQDGGRARPTG*AGFRVW H*KGPAGPGLHFRLGIWEWRERGGLLLVRWLHQQ HLHHLRQQRHREWLQALVPGRVCLHPGHHLQQW GLL*AKNRHHGSASALYRWPHWDLSLCPHGGGH HRLGSRSKEHPLSAGAADYGPDQRLRGALGPAGG LLGARGGSHLHLTPTPRRPPDLPGFSLGNQVST SGKEVAGSFQ*RVYKLGIHDCPLLGRKG*RAVD LGNPRSAIPGPQPGEAREVERMEPHTVWHSRAPV PHLQCPSVPLADAGALSPRAGATQGCPVTLPGG SS*R*GRLHSSIHPRLC*YFTDQCVPSGVW*QR L*WPQCRPVLELRPLQPGECQDQQEVRECVPLGL LWGHSSKTLSPVPQGV*DLLQQSCDAVPVLPPR VLSPPGDEHLCDPLSCRILC**KSEKLP*MPPK L*KVRG*T*EMYCL*RRIQPCTGQLHS*L*ARHL L*LRADQMWGMPSHLRNLRGARQRRVHSLCEKL PLPRLEVCASLW*GLLPRRDAGLAPQSVSKV*R ELLELCRLQQEL*QV*DGLHTAGDLLHHQPHVQQ R*RDILRDGEVQPAVRTEALHSVLLPHVPPGRV RVPSCPQRAGTPIHPSVHLPPDCRPESVSGAAP CT*QLYLPRSSISEHPSQVGGGS*GGVPKMVISS QMLLVGSSLPTN*QEQNEFWESTALALEQLLGP *VY*IFKTKAEKKGAWHHTSLFSPCFSAAV**I SPAQLANPGPSSHVTKGQQSTCSLPATEQSGDGL VRL*IR*V*GHKMYDHWGRSIYFYIVSYF*NCS SGLESPIPKPDQKILSPAALSFEQAELSLLPCS VCVFRSLMA*TTTST*CRAF*GEATGGILWPAKR YPDGQAIPLNKM*SQPCIVNHPSLPILWNCF*N TFGLCPSEDSFLRWKLLCLCILQAYMTQLDLFC QTCVGIL*ATCSNFYRC*LF*QIFHIFSLKCTDL LDQFP*IGK*HLP*IFSTSSFSILSCSPV*RQC ICLVTCELERTQMLFIESTTLKEKSMKQVQC*P *INKRVNIPWH

Frame 1

PGPIGNLEDYYHFYHSKTFKRSTLSSRGPHTFL RMDPQVKWLQQQEVKRRVKRQVRSDPQALYFND PIWSNMWYLHCGDKNSRCRSEMNVQAAWKRGYTG KNVVVTILDDGIERNHPDLAPNYDSYASYDVNG NDYDPSPRYDASNENKHGTRCAGEVAASANNSY CIVGIAYNAKIGGIRMLDGDVTDVVEAKSLGIRP NYIDIYSASWGPDDDGKTVDGPGRLAKQAFEYG IKKGRQGLGSIFVWASGNGGREGDYCSCDGYTN SIYTISVSSATENGYKPWYLEECASTLATTYSSG AFYERKIVTTDLRQRCTDGHTGTSVSAPMVAGI IALALEAKSIPLVQVLRTTALTSACAEHSDQRV VYLEHVVVRTSISHPRRGDLQIYLVSPSGTKSQL LAKRLLDLSNEGFTNWEFMTVHCWGEKAEGQWT LEIQDLPSQVRNPEKQGKLKEWSLILYGTAEHP YHTFSAHQSRSRMLELSAPELEPPKAALSPSQVE VPEDEEDYTAQSTPGSANILQTSVCHPECGDKG CDGPNADQCLNCVHFSLGSVKTSRKCVSVCPLG YFGDTAARRCRRCHKGCETCSSRAATQCLSCRRG FYHHQEMNTCVTLCPAGFYADESQKNCLKCHPS CKKCVDEPEKCTVCKEGFSLARGSCIPDCEPGT YFDSELIRCGECHHTCGTCVGPGREECIHCAKNF HFHDWKCVPACGEGFYPEEMPGLPHKVCRRCDE NCLSCAGSSRNCSRCKTGFTQLGTSCITNHTCS NADETFCEMVKSNRLCERKLFIQFCCRTCLLAG* GCLAAHRGQALPSIHPSTFLQTVGQSLFQERRP APDSFISPGAASLSTQARWVVALKEVFLKW*YP LKCCLLAPVFRQTNRNKMNSGNPQLWLWSSFWDH KFTESSRPKQKRKALGITHHSSPRAFLRLCSKS PRPSWRTLGHPHM*QRASSLPARCLPLSSLGTV WSDYK*DRFEGIKCMTTGAGVSIST*SATSETAA VA*KVQFQSQTRRFYPPQRSPLSKPSSPCYRVL SVSSGVSWPERPPRPDAEPSEERQQEAFCGQPK GTPMAKQFL*TKCKASHALLIIHHFPFYGIAFKI HLASALQKTRF*GGNSCVCVYYKPT*HSWIYSA KPV*AFYKLHVLIFTDVNYFDKYFIYFH*NAQI CLINSLE*GSNICLKFFRPRLSPYCPAPLFDDSA FALSPVSWREPRCCLLNLQL*KRNQ*SKYNVNP KLIKELTSHG

Frame 2

LGQLETWKITTIFITAKPLKDQP*VAEALTPSS EWTPR*NGSSNRK*NEG*RDRCEVTRRPFTSTT PFGPTCGTCIVATRTVAAGRK*MSRQRGRGATQ EKTWWSPSLMMA*REITLTWPQIMIPTPATT*TA MIMTHLHDMMPAMKINTALVVREKLLLQQTIPT ASWA*RTMPK*EASACWTAMSQMWSRQSRWASD PTTSTFTVPAGGRTTTARRWTGPADWLSRLSSMA LKRAGRAWAPFSSGHLGMAGERGTTARAMATPT ASTPSPSAAPPRMATSPGTWKSVPPPWPPPTAV GPFMSEKSSPRICVSAVPMATLGPQSLPPWWRAS SPWL*KQRASP*CRCCGLRP*PAPARSTRTSGW STWSTWWFAPPSHTHAEETSRSTWFLPREPSLN FWQRGCWIFPMKGLQTGNS*LSTAGEKRLKGSGP WKSKICHPRSATRRSKGS*KNGASYCMAQQSTR TTPSVPISPARGCWSSQPQSWSHPRLPCHPPRW KFLKMRKITQLNPPQALLIFYRPVCAIRSVVTKA VMAPMQTSA*TASTSAWGVSRPAGSA*VCAPWA TLGTQQQDAVAGATRGVRPAPAELRRSACLAAA GSITTRR*TPV*PSVLQDFMLMKVRKIALNATQA VKSAWMNLRNVLSVKKDSALHGAAAFLTVSQAP TLTQS*SDVGNAITPAEPAWGQAEKSAFTVRKT STSTTGSVCQPVVRASTQKRCRACPTKCVEGVTR TA*AVQAPAGTVAGVRRASHSWGPPASPTTRAA TLTRHSARW*SPTGCANGSSSFSSAAARASWPG KGA*LPTEGRHSHPSIRPPSSRLSARVCFRSGAL HLTALSPQEQHL*APKPGGWWLLRRCS*NGDIL SNAACWLQSSDKLTGTK*ILGIHSSGFGAASGT ISLLNLQDQSRKERRLASHITLLPVLFCGCVVNL PGPAGEPWAILTCDKGPAVYLLVACH*AVWGRF GQTINKIGLRA*NV*PLGPEYLFLHSQLLLKLQ QWLRKSNSKARPEDSIPRSALL*ASRALLVTVFC LCLQESHGLNDHLDLMQSLLRRGNRRHSVASQK VPRWPSNSSEQNVKPAMHC*SSITSHFMELLLK YIWPLPFRRLVFKVETPVSVYITSLHDTVGFILP NLCRHFISYMF*FLPMLIILTNISYIFIEMHRS A*SIPLNREVTFALNFFDLVFLHIVLLPCLTTV HLPCHL*AGENPDVVY*IYNSEREINEASTMLTL N**KS*HPMA

Sequence PCSK6.dAug05

TCGCGGGCCGAGGACGCCTCTGGGGCGGCACCGCGTCCCGAGAGCCCCAGAAGTCGGCGGGGAAGTTCCCCCGGTGGGGGGCGTTTCGGGCCTCCCGGAC GGCTCTCGGCCCCGGAGCCCGGTCGCAGGAGCGCGGGCCCGGGGGCGGGAACGCGCCGCGGCCGCCTCCTCCTCCCCGGCTCCCGCCCGCGGCGGTGTTG GCGGCGGCGGTGGCGGCGGCGGCGGCGCTTCCCCGGCGCGGAGCGGCTTTAAAAGGCGGCACTCCACCCCCCGGCGCACTCGCAGCTCGGGCGCCGCGCG AGCCTGTCGCCGCTATGCCTCCGCGCGCGCCGCCTGCGCCCGGGCCCCGGCCGCCGCCCCGGGCCGCCGCCGCCACCGACACCGCCGCGGGCGCGGGGGG CGCGGGGGGCGCGGGGGGCGCCGGCGGGCCCGGGTTCCGGCCGCTCGCGCCGCGTCCCTGGCGCTGGCTGCTGCTGCTGGCGCTGCCTGCCGCCTGCTCC GCCCCCGCCGCGCCCCGTCTACACCAACCACTGGGCGGTGCAAGTGCTGGGCGGCCCGGCCGAGGCGGACCGCGTGGCGGCGGCGCACGGGTACCTCAAC TTGGGCCAGATTGGAAACCTGGAAGATTACTACCATTTTTATCACAGCAAAACCTTTAAAAGATCAACCTTGAGTAGCAGAGGCCCTCACACCTTCCTCA GAATGGACCCCCAGGTGAAATGGCTCCAGCAACAGGAAGTGAAACGAAGGGTGAAGAGACAGGTGCGAAGTGACCCGCAGGCCCTTTACTTCAACGACCC CATTTGGTCCAACATGTGGTACCTGCATTGTGGCGACAAGAACAGTCGCTGCCGGTCGGAAATGAATGTCCAGGCAGCGTGGAAGAGGGGCTACACAGGA AAAAACGTGGTGGTCACCATCCTTGATGATGGCATAGAGAGAAATCACCCTGACCTGGCCCCAAATTATGATTCCTACGCCAGCTACGACGTGAACGGCA ATGATTATGACCCATCTCCACGATATGATGCCAGCAATGAAAATAAACACGGCACTCGTTGTGCGGGAGAAGTTGCTGCTTCAGCAAACAATTCCTACTG CATCGTGGGCATAGCGTACAATGCCAAAATAGGAGGCATCCGCATGCTGGACGGCGATGTCACAGATGTGGTCGAGGCAAAGTCGCTGGGCATCAGACCC AACTACATCGACATTTACAGTGCCAGCTGGGGGCCGGACGACGACGGCAAGACGGTGGACGGGCCCGGCCGACTGGCTAAGCAGGCTTTCGAGTATGGCA TTAAAAAGGGCCGGCAGGGCCTGGGCTCCATTTTCGTCTGGGCATCTGGGAATGGCGGGAGAGAGGGGGACTACTGCTCGTGCGATGGCTACACCAACAG CATCTACACCATCTCCGTCAGCAGCGCCACCGAGAATGGCTACAAGCCCTGGTACCTGGAAGAGTGTGCCTCCACCCTGGCCACCACCTACAGCAGTGGG GCCTTTTATGAGCGAAAAATCGTCACCACGGATCTGCGTCAGCGCTGTACCGATGGCCACACTGGGACCTCAGTCTCTGCCCCCATGGTGGCGGGCATCA TCGCCTTGGCTCTAGAAGCAAACAGCCAGTTAACCTGGAGGGACGTCCAGCACCTGCTAGTGAAGACATCCCGGCCGGCCCACCTGAAAGCGAGCGACTG GAAAGTGAACGGCGCGGGTCATAAAGTTAGCCATTTCTATGGATTTGGTTTGGTGGACGCAGAAGCTCTCGTTGTGGAGGCAAAGAAGTGGACAGCAGTG CCATCGCAGCACATGTGTGTGGCCGCCTCGGACAAGAGACCCAGGAGCATCCCCTTAGTGCAGGTGCTGCGGACTACGGCCCTGACCAGCGCCTGCGCGG AGCACTCGGACCAGCGGGTGGTCTACTTGGAGCACGTGGTGGTTCGCACCTCCATCTCACACCCACGCCGAGGAGACCTCCAGATCTACCTGGTTTCTCC CTCGGGAACCAAGTCTCAACTTCTGGCAAAGAGGTTGCTGGATCTTTCCAATGAAGGGTTTACAAACTGGGAATTCATGACTGTCCACTGCTGGGGAGAA AAGGCTGAAGGGCAGTGGACCTTGGAAATCCAAGATCTGCCATCCCAGGTCCGCAACCCGGAGAAGCAAGGGAAGTTGAAAGAATGGAGCCTCATACTGT ATGGCACAGCAGAGCACCCGTACCACACCTTCAGTGCCCATCAGTCCCGCTCGCGGATGCTGGAGCTCTCAGCCCCAGAGCTGGAGCCACCCAAGGCTGC CCTGTCACCCTCCCAGGTGGAAGTTCCTGAAGATGAGGAAGATTACACAGGTGTGTGCCATCCGGAGTGTGGTGACAAAGGCTGTGATGGCCCCAATGCA GACCAGTGCTTGAACTGCGTCCACTTCAGCCTGGGGAGTGTCAAGACCAGCAGGAAGTGCGTGAGTGTGTGCCCCTTGGGCTACTTTGGGGACACAGCAG CAAGACGCTGTCGCCGGTGCCACAAGGGGTGTGAGACCTGCTCCAGCAGAGCTGCGACGCAGTGCCTGTCTTGCCGCCGCGGGTTCTATCACCACCAGGA GATGAACACCTGTGTGACCCTCTGTCCTGCAGGATTTTATGCTGATGAAAGTCAGAAAAATTGCCTTAAATGCCACCCAAGCTGTAAAAAGTGCGTGGAT GAACCTGAGAAATGTACTGTCTGTAAAGAAGGATTCAGCCTTGCACGGGGCAGCTGCATTCCTGACTGTGAGCCAGGCACCTACTTTGACTCAGAGCTGA TCAGATGTGGGGAATGCCATCACACCTGCGGAACCTGCGTGGGGCCAGGCAGAGAAGAGTGCATTCACTGTGCGAAAAACTTCCACTTCCACGACTGGAA GTGTGTGCCAGCCTGTGGTGAGGGCTTCTACCCAGAAGAGATGCCGGGCTTGCCCCACAAAGTGTGTCGAAGGTGTGACGAGAACTGCTTGAGCTGTGCA GGCTCCAGCAGGAACTGTAGCAGGTGTAAGACGGGCTTCACACAGCTGGGGACCTCCTGCATCACCAACCACACGTGCAGCAACGCTGACGAGACATTCT GCGAGATGGTGAAGTCCAACCGGCTGTGCGAACGGAAGCTCTTCATTCAGTTCTGCTGCCGCACGTGCCTCCTGGCCGGGTAAGGGTGCCTAGCTGCCCA CAGAGGGCAGGCACTCCCATCCATCCATCCGTCCACCTTCCTCCAGACTGTCGGCCAGAGTCTGTTTCAGGAGCGGCGCCCTGCACCTGACAGCTTTATC TCCCCAGGAGCAGCATCTCTGAGCACCCAAGCCAGGTGGGTGGTGGCTCTTAAGGAGGTGTTCCTAAAATGGTGATATCCTCTCAAATGCTGCTTGTTGG CTCCAGTCTTCCGACAAACTAACAGGAACAAAATGAATTCTGGGAATCCACAGCTCTGGCTTTGGAGCAGCTTCTGGGACCATAAGTTTACTGAATCTTC AAGACCAAAGCAGAAAAGAAAGGCGCTTGGCATCACACATCACTCTTCTCCCCGTGCTTTTCTGCGGCTGTGTAGTAAATCTCCCCGGCCCAGCTGGCGA ACCCTGGGCCATCCTCACATGTGACAAAGGGCCAGCAGTCTACCTGCTCGTTGCCTGCCACTGAGCAGTCTGGGGACGGTTTGGTCAGACTATAAATAAG ATAGGTTTGAGGGCATAAAATGTATGACCACTGGGGCCGGAGTATCTATTTCTACATAGTCAGCTACTTCTGAAACTGCAGCAGTGGCTTAGAAAGTCCA ATTCCAAAGCCAGACCAGAAGATTCTATCCCCCGCAGCGCTCTCCTTTGAGCAAGCCGAGCTCTCCTTGTTACCGTGTTCTGTCTGTGTCTTCAGGAGTC TCATGGCCTGAACGACCACCTCGACCTGATGCAGAGCCTTCTGAGGAGAGGCAACAGGAGGCATTCTGTGGCCAGCCAAAAGGTACCCCGATGGCCAAGC AATTCCTCTGAACAAAATGTAAAGCCAGCCATGCATTGTTAATCATCCATCACTTCCCATTTTATGGAATTGCTTTTAAAATACATTTGGCCTCTGCCCT TCAGAAGACTCGTTTTTAAGGTGGAAACTCCTGTGTCTGTGTATATTACAAGCCTACATGACACAGTTGGATTTATTCTGCCAAACCTGTGTAGGCATTT TATAAGCTACATGTTCTAATTTTTACCGATGTTAATTATTTTGACAAATATTTCATATATTTTCATTGAAATGCACAGATCTGCTTGATCAATTCCCTTG AATAGGGAAGTAACATTTGCCTTAAATTTTTTCGACCTCGTCTTTCTCCATATTGTCCTGCTCCCCTGTTTGACGACAGTGCATTTGCCTTGTCACCTGT GAGCTGGAGAGAACCCAGATGTTGTTTATTGAATCTACAACTCTGAAAGAGAAATCAATGAAGCAAGTACAATGTTAACCCTAAATTAATAAAAGAGTTA ACATCCCATGGCAC

PCSK6.F1 (forward primer)
PCSK6.R33 (reverse primer)
Single exon skipping

Frame 0

SRAEDASGAAPRPESPRSRRGSSPGGGRFGPPG RLSAPEPGRRSAGPGAGTRRGRLLLPGSRPRRCW RRRWRRRRRFPGAERL*KAALHPPAHSQLGRRA SLSPLCLRARRLRPGPGRRPGPPPPPTPPRARG ARGARGAPAGPGSGRSRRVPGAGCCCWRCLPPAP PPPRPVYTNHWAVQVLGGPAEADRVAAAHGYLN LGQIGNLEDYYHFYHSKTFKRSTLSSRGPHTFL RMDPQVKWLQQQEVKRRVKRQVRSDPQALYFNDP IWSNMWYLHCGDKNSRCRSEMNVQAAWKRGYTG KNVVVTILDDGIERNHPDLAPNYDSYASYDVNG NDYDPSPRYDASNENKHGTRCAGEVAASANNSYC IVGIAYNAKIGGIRMLDGDVTDVVEAKSLGIRP NYIDIYSASWGPDDDGKTVDGPGRLAKQAFEYG IKKGRQGLGSIFVWASGNGGREGDYCSCDGYTNS IYTISVSSATENGYKPWYLEECASTLATTYSSG AFYERKIVTTDLRQRCTDGHTGTSVSAPMVAGI IALALEANSQLTWRDVQHLLVKTSRPAHLKASDW KVNGAGHKVSHFYGFGLVDAEALVVEAKKWTAV PSQHMCVAASDKRPRSIPLVQVLRTTALTSACA EHSDQRVVYLEHVVVRTSISHPRRGDLQIYLVSP SGTKSQLLAKRLLDLSNEGFTNWEFMTVHCWGE KAEGQWTLEIQDLPSQVRNPEKQGKLKEWSLIL YGTAEHPYHTFSAHQSRSRMLELSAPELEPPKAA LSPSQVEVPEDEEDYTGVCHPECGDKGCDGPNA DQCLNCVHFSLGSVKTSRKCVSVCPLGYFGDTA ARRCRRCHKGCETCSSRAATQCLSCRRGFYHHQE MNTCVTLCPAGFYADESQKNCLKCHPSCKKCVD EPEKCTVCKEGFSLARGSCIPDCEPGTYFDSEL IRCGECHHTCGTCVGPGREECIHCAKNFHFHDWK CVPACGEGFYPEEMPGLPHKVCRRCDENCLSCA GSSRNCSRCKTGFTQLGTSCITNHTCSNADETF CEMVKSNRLCERKLFIQFCCRTCLLAG*GCLAAH RGQALPSIHPSTFLQTVGQSLFQERRPAPDSFI SPGAASLSTQARWVVALKEVFLKW*YPLKCCLL APVFRQTNRNKMNSGNPQLWLWSSFWDHKFTESS RPKQKRKALGITHHSSPRAFLRLCSKSPRPSWR TLGHPHM*QRASSLPARCLPLSSLGTVWSDYK* DRFEGIKCMTTGAGVSIST*SATSETAAVA*KVQ FQSQTRRFYPPQRSPLSKPSSPCYRVLSVSSGV SWPERPPRPDAEPSEERQQEAFCGQPKGTPMAK QFL*TKCKASHALLIIHHFPFYGIAFKIHLASAL QKTRF*GGNSCVCVYYKPT*HSWIYSAKPV*AF YKLHVLIFTDVNYFDKYFIYFH*NAQICLINSL E*GSNICLKFFRPRLSPYCPAPLFDDSAFALSPV SWREPRCCLLNLQL*KRNQ*SKYNVNPKLIKEL TSHG

Frame 1

RGPRTPLGRHRVPRAPEVGGEVPPVGGVSGLPD GSRPRSPVAGARARGRERAAAASSSPAPARGGV GGGGGGGGGASPARSGFKRRHSTPRRTRSSGAAR ACRRYASARAACARAPAAAPGRRRHRHRRGRGG RGGRGGRRRARVPAARAASLALAAAAGAACRLL RPRRAPSTPTTGRCKCWAARPRRTAWRRRTGTST WARLETWKITTIFITAKPLKDQP*VAEALTPSS EWTPR*NGSSNRK*NEG*RDRCEVTRRPFTSTT PFGPTCGTCIVATRTVAAGRK*MSRQRGRGATQE KTWWSPSLMMA*REITLTWPQIMIPTPATT*TA MIMTHLHDMMPAMKINTALVVREKLLLQQTIPT ASWA*RTMPK*EASACWTAMSQMWSRQSRWASDP TTSTFTVPAGGRTTTARRWTGPADWLSRLSSMA LKRAGRAWAPFSSGHLGMAGERGTTARAMATPT ASTPSPSAAPPRMATSPGTWKSVPPPWPPPTAVG PFMSEKSSPRICVSAVPMATLGPQSLPPWWRAS SPWL*KQTAS*PGGTSSTC**RHPGRPT*KRAT GK*TARVIKLAISMDLVWWTQKLSLWRQRSGQQC HRSTCVWPPRTRDPGASP*CRCCGLRP*PAPAR STRTSGWSTWSTWWFAPPSHTHAEETSRSTWFL PREPSLNFWQRGCWIFPMKGLQTGNS*LSTAGEK RLKGSGPWKSKICHPRSATRRSKGS*KNGASYC MAQQSTRTTPSVPISPARGCWSSQPQSWSHPRL PCHPPRWKFLKMRKITQVCAIRSVVTKAVMAPMQ TSA*TASTSAWGVSRPAGSA*VCAPWATLGTQQ QDAVAGATRGVRPAPAELRRSACLAAAGSITTR R*TPV*PSVLQDFMLMKVRKIALNATQAVKSAWM NLRNVLSVKKDSALHGAAAFLTVSQAPTLTQS* SDVGNAITPAEPAWGQAEKSAFTVRKTSTSTTG SVCQPVVRASTQKRCRACPTKCVEGVTRTA*AVQ APAGTVAGVRRASHSWGPPASPTTRAATLTRHS ARW*SPTGCANGSSSFSSAAARASWPGKGA*LP TEGRHSHPSIRPPSSRLSARVCFRSGALHLTALS PQEQHL*APKPGGWWLLRRCS*NGDILSNAACW LQSSDKLTGTK*ILGIHSSGFGAASGTISLLNL QDQSRKERRLASHITLLPVLFCGCVVNLPGPAGE PWAILTCDKGPAVYLLVACH*AVWGRFGQTINK IGLRA*NV*PLGPEYLFLHSQLLLKLQQWLRKS NSKARPEDSIPRSALL*ASRALLVTVFCLCLQES HGLNDHLDLMQSLLRRGNRRHSVASQKVPRWPS NSSEQNVKPAMHC*SSITSHFMELLLKYIWPLP FRRLVFKVETPVSVYITSLHDTVGFILPNLCRHF ISYMF*FLPMLIILTNISYIFIEMHRSA*SIPL NREVTFALNFFDLVFLHIVLLPCLTTVHLPCHL *AGENPDVVY*IYNSEREINEASTMLTLN**KS* HPMA

Frame 2

AGRGRLWGGTASREPQKSAGKFPRWGAFRASRT ALGPGARSQERGPGGGNAPRPPPPPRLPPAAVL AAAVAAAAALPRRGAALKGGTPPPGALAARAPR EPVAAMPPRAPPAPGPRPPPRAAAATDTAAGAGG AGGAGGAGGPGFRPLAPRPWRWLLLLALPAACS APAAPRLHQPLGGASAGRPGRGGPRGGGARVPQ LGPDWKPGRLLPFLSQQNL*KINLE*QRPSHLPQ NGPPGEMAPATGSETKGEETGAK*PAGPLLQRP HLVQHVVPALWRQEQSLPVGNECPGSVEEGLHR KKRGGHHP**WHREKSP*PGPKL*FLRQLRRERQ *L*PISTI*CQQ*K*TRHSLCGRSCCFSKQFLL HRGHSVQCQNRRHPHAGRRCHRCGRGKVAGHQT QLHRHLQCQLGAGRRRQDGGRARPTG*AGFRVWH *KGPAGPGLHFRLGIWEWRERGGLLLVRWLHQQ HLHHLRQQRHREWLQALVPGRVCLHPGHHLQQW GLL*AKNRHHGSASALYRWPHWDLSLCPHGGGHH RLGSRSKQPVNLEGRPAPASEDIPAGPPESERL ESERRGS*S*PFLWIWFGGRRSSRCGGKEVDSS AIAAHVCGRLGQETQEHPLSAGAADYGPDQRLRG ALGPAGGLLGARGGSHLHLTPTPRRPPDLPGFS LGNQVSTSGKEVAGSFQ*RVYKLGIHDCPLLGR KG*RAVDLGNPRSAIPGPQPGEAREVERMEPHTV WHSRAPVPHLQCPSVPLADAGALSPRAGATQGC PVTLPGGSS*R*GRLHRCVPSGVW*QRL*WPQC RPVLELRPLQPGECQDQQEVRECVPLGLLWGHSS KTLSPVPQGV*DLLQQSCDAVPVLPPRVLSPPG DEHLCDPLSCRILC**KSEKLP*MPPKL*KVRG *T*EMYCL*RRIQPCTGQLHS*L*ARHLL*LRAD QMWGMPSHLRNLRGARQRRVHSLCEKLPLPRLE VCASLW*GLLPRRDAGLAPQSVSKV*RELLELC RLQQEL*QV*DGLHTAGDLLHHQPHVQQR*RDIL RDGEVQPAVRTEALHSVLLPHVPPGRVRVPSCP QRAGTPIHPSVHLPPDCRPESVSGAAPCT*QLY LPRSSISEHPSQVGGGS*GGVPKMVISSQMLLVG SSLPTN*QEQNEFWESTALALEQLLGP*VY*IF KTKAEKKGAWHHTSLFSPCFSAAV**ISPAQLA NPGPSSHVTKGQQSTCSLPATEQSGDGLVRL*IR *V*GHKMYDHWGRSIYFYIVSYF*NCSSGLESP IPKPDQKILSPAALSFEQAELSLLPCSVCVFRS LMA*TTTST*CRAF*GEATGGILWPAKRYPDGQA IPLNKM*SQPCIVNHPSLPILWNCF*NTFGLCP SEDSFLRWKLLCLCILQAYMTQLDLFCQTCVGI L*ATCSNFYRC*LF*QIFHIFSLKCTDLLDQFP* IGK*HLP*IFSTSSFSILSCSPV*RQCICLVTC ELERTQMLFIESTTLKEKSMKQVQC*P*INKRV NIPWH

Sequence PCSK6.cAug05

TCGCGGGCCGAGGACGCCTCTGGGGCGGCACCGCGTCCCGAGAGCCCCAGAAGTCGGCGGGGAAGTTCCCCCGGTGGGGGGCGTTTCGGGCCTCCCGGAC GGCTCTCGGCCCCGGAGCCCGGTCGCAGGAGCGCGGGCCCGGGGGCGGGAACGCGCCGCGGCCGCCTCCTCCTCCCCGGCTCCCGCCCGCGGCGGTGTTG GCGGCGGCGGTGGCGGCGGCGGCGGCGCTTCCCCGGCGCGGAGCGGCTTTAAAAGGCGGCACTCCACCCCCCGGCGCACTCGCAGCTCGGGCGCCGCGCG AGCCTGTCGCCGCTATGCCTCCGCGCGCGCCGCCTGCGCCCGGGCCCCGGCCGCCGCCCCGGGCCGCCGCCGCCACCGACACCGCCGCGGGCGCGGGGGG CGCGGGGGGCGCGGGGGGCGCCGGCGGGCCCGGGTTCCGGCCGCTCGCGCCGCGTCCCTGGCGCTGGCTGCTGCTGCTGGCGCTGCCTGCCGCCTGCTCC GCCCCCGCCGCGCCCCGTCTACACCAACCACTGGGCGGTGCAAGTGCTGGGCGGCCCGGCCGAGGCGGACCGCGTGGCGGCGGCGCACGGGTACCTCAAC TTGGGCCAGATTGGAAACCTGGAAGATTACTACCATTTTTATCACAGCAAAACCTTTAAAAGATCAACCTTGAGTAGCAGAGGCCCTCACACCTTCCTCA GAATGGACCCCCAGGTGAAATGGCTCCAGCAACAGGAAGTGAAACGAAGGGTGAAGAGACAGGTGCGAAGTGACCCGCAGGCCCTTTACTTCAACGACCC CATTTGGTCCAACATGTGGTACCTGCATTGTGGCGACAAGAACAGTCGCTGCCGGTCGGAAATGAATGTCCAGGCAGCGTGGAAGAGGGGCTACACAGGA AAAAACGTGGTGGTCACCATCCTTGATGATGGCATAGAGAGAAATCACCCTGACCTGGCCCCAAATTATGATTCCTACGCCAGCTACGACGTGAACGGCA ATGATTATGACCCATCTCCACGATATGATGCCAGCAATGAAAATAAACACGGCACTCGTTGTGCGGGAGAAGTTGCTGCTTCAGCAAACAATTCCTACTG CATCGTGGGCATAGCGTACAATGCCAAAATAGGAGGCATCCGCATGCTGGACGGCGATGTCACAGATGTGGTCGAGGCAAAGTCGCTGGGCATCAGACCC AACTACATCGACATTTACAGTGCCAGCTGGGGGCCGGACGACGACGGCAAGACGGTGGACGGGCCCGGCCGACTGGCTAAGCAGGCTTTCGAGTATGGCA TTAAAAAGGGCCGGCAGGGCCTGGGCTCCATTTTCGTCTGGGCATCTGGGAATGGCGGGAGAGAGGGGGACTACTGCTCGTGCGATGGCTACACCAACAG CATCTACACCATCTCCGTCAGCAGCGCCACCGAGAATGGCTACAAGCCCTGGTACCTGGAAGAGTGTGCCTCCACCCTGGCCACCACCTACAGCAGTGGG GCCTTTTATGAGCGAAAAATCGTCACCACGGATCTGCGTCAGCGCTGTACCGATGGCCACACTGGGACCTCAGTCTCTGCCCCCATGGTGGCGGGCATCA TCGCCTTGGCTCTAGAAGCAAACAGCCAGTTAACCTGGAGGGACGTCCAGCACCTGCTAGTGAAGACATCCCGGCCGGCCCACCTGAAAGCGAGCGACTG GAAAGTGAACGGCGCGGGTCATAAAGTTAGCCATTTCTATGGATTTGGTTTGGTGGACGCAGAAGCTCTCGTTGTGGAGGCAAAGAAGTGGACAGCAGTG CCATCGCAGCACATGTGTGTGGCCGCCTCGGACAAGAGACCCAGGAGCATCCCCTTAGTGCAGGTGCTGCGGACTACGGCCCTGACCAGCGCCTGCGCGG AGCACTCGGACCAGCGGGTGGTCTACTTGGAGCACGTGGTGGTTCGCACCTCCATCTCACACCCACGCCGAGGAGACCTCCAGATCTACCTGGTTTCTCC CTCGGGAACCAAGTCTCAACTTCTGGCAAAGAGGTTGCTGGATCTTTCCAATGAAGGGTTTACAAACTGGGAATTCATGACTGTCCACTGCTGGGGAGAA AAGGCTGAAGGGCAGTGGACCTTGGAAATCCAAGATCTGCCATCCCAGGTCCGCAACCCGGAGAAGCAAGGGAAGTTGAAAGAATGGAGCCTCATACTGT ATGGCACAGCAGAGCACCCGTACCACACCTTCAGTGCCCATCAGTCCCGCTCGCGGATGCTGGAGCTCTCAGCCCCAGAGCTGGAGCCACCCAAGGCTGC CCTGTCACCCTCCCAGGTGGAAGTTCCTGAAGATGAGGAAGATTACACAGGTGTGTGCCATCCGGAGTGTGGTGACAAAGGCTGTGATGGCCCCAATGCA GACCAGTGCTTGAACTGCGTCCACTTCAGCCTGGGGAGTGTCAAGACCAGCAGGAAGTGCGTGAGTGTGTGCCCCTTGGGCTACTTTGGGGACACAGCAG CAAGACGCTGTCGCCGGTGCCACAAGGGGTGTGAGACCTGCTCCAGCAGAGCTGCGACGCAGTGCCTGTCTTGCCGCCGCGGGTTCTATCACCACCAGGA GATGAACACCTGTGTGACCCTCTGTCCTGCAGGATTTTATGCTGATGAAAGTCAGAAAAATTGCCTTAAATGCCACCCAAGCTGTAAAAAGTGCGTGGAT GAACCTGAGAAATGTACTGTCTGTAAAGAAGGATTCAGCCTTGCACGGGGCAGCTGCATTCCTGACTGTGAGCCAGGCACCTACTTTGACTCAGAGCTGA TCAGATGTGGGGAATGCCATCACACCTGCGGAACCTGCGTGGGGCCAGGCAGAGAAGAGTGCATTCACTGTGCGAAAAACTTCCACTTCCACGACTGGAA GTGTGTGCCAGCCTGTGGTGAGGGCTTCTACCCAGAAGAGATGCCGGGCTTGCCCCACAAAGTGTGTCGAAGGTACGGTCCTCCTGGCGGGGAACGGCAG GCAGCTGTGTCCAGCAAAGGAGTGCCTGGAGGGCAGAGTCTGGCAGCATCTTCCCCAGGGGCCGGGGAGGGCATGTTGCATCACCCCACTGTGGACAGGA GCCCATTTACAGAGCTGCTGAGGGGCCTTCGTCCCTTTGTTCACTGGATGCATATTTGTTGGGTCCCTGCTGTGGGCAGACACCGTGCAGCAGCGGGATA AGATGGGAAGCAGAATAGCAAGGTGCTCACACCCCATTGGTGGACTACGCGGGACGTCCCTGCCCATGTCGCGCTCAGCATGCATCCTTTGCACTGTCGA TAATCAGAGAAACCATGTTTTGGGTTAAAAA

PCSK6.F1 (forward primer)
PCSK6.R33 (reverse primer)
Single exon skipping

Frame 0

SRAEDASGAAPRPESPRSRRGSSPGGGRFGPPG RLSAPEPGRRSAGPGAGTRRGRLLLPGSRPRRCW RRRWRRRRRFPGAERL*KAALHPPAHSQLGRRA SLSPLCLRARRLRPGPGRRPGPPPPPTPPRARG ARGARGAPAGPGSGRSRRVPGAGCCCWRCLPPAP PPPRPVYTNHWAVQVLGGPAEADRVAAAHGYLN LGQIGNLEDYYHFYHSKTFKRSTLSSRGPHTFL RMDPQVKWLQQQEVKRRVKRQVRSDPQALYFNDP IWSNMWYLHCGDKNSRCRSEMNVQAAWKRGYTG KNVVVTILDDGIERNHPDLAPNYDSYASYDVNG NDYDPSPRYDASNENKHGTRCAGEVAASANNSYC IVGIAYNAKIGGIRMLDGDVTDVVEAKSLGIRP NYIDIYSASWGPDDDGKTVDGPGRLAKQAFEYG IKKGRQGLGSIFVWASGNGGREGDYCSCDGYTNS IYTISVSSATENGYKPWYLEECASTLATTYSSG AFYERKIVTTDLRQRCTDGHTGTSVSAPMVAGI IALALEANSQLTWRDVQHLLVKTSRPAHLKASDW KVNGAGHKVSHFYGFGLVDAEALVVEAKKWTAV PSQHMCVAASDKRPRSIPLVQVLRTTALTSACA EHSDQRVVYLEHVVVRTSISHPRRGDLQIYLVSP SGTKSQLLAKRLLDLSNEGFTNWEFMTVHCWGE KAEGQWTLEIQDLPSQVRNPEKQGKLKEWSLIL YGTAEHPYHTFSAHQSRSRMLELSAPELEPPKAA LSPSQVEVPEDEEDYTGVCHPECGDKGCDGPNA DQCLNCVHFSLGSVKTSRKCVSVCPLGYFGDTA ARRCRRCHKGCETCSSRAATQCLSCRRGFYHHQE MNTCVTLCPAGFYADESQKNCLKCHPSCKKCVD EPEKCTVCKEGFSLARGSCIPDCEPGTYFDSEL IRCGECHHTCGTCVGPGREECIHCAKNFHFHDWK CVPACGEGFYPEEMPGLPHKVCRRYGPPGGERQ AAVSSKGVPGGQSLAASSPGAGEGMLHHPTVDR SPFTELLRGLRPFVHWMHICWVPAVGRHRAAAG* DGKQNSKVLTPHWWTTRDVPAHVALSMHPLHCR *SEKPCFGLK

Frame 1

RGPRTPLGRHRVPRAPEVGGEVPPVGGVSGLPD GSRPRSPVAGARARGRERAAAASSSPAPARGGV GGGGGGGGGASPARSGFKRRHSTPRRTRSSGAAR ACRRYASARAACARAPAAAPGRRRHRHRRGRGG RGGRGGRRRARVPAARAASLALAAAAGAACRLL RPRRAPSTPTTGRCKCWAARPRRTAWRRRTGTST WARLETWKITTIFITAKPLKDQP*VAEALTPSS EWTPR*NGSSNRK*NEG*RDRCEVTRRPFTSTT PFGPTCGTCIVATRTVAAGRK*MSRQRGRGATQE KTWWSPSLMMA*REITLTWPQIMIPTPATT*TA MIMTHLHDMMPAMKINTALVVREKLLLQQTIPT ASWA*RTMPK*EASACWTAMSQMWSRQSRWASDP TTSTFTVPAGGRTTTARRWTGPADWLSRLSSMA LKRAGRAWAPFSSGHLGMAGERGTTARAMATPT ASTPSPSAAPPRMATSPGTWKSVPPPWPPPTAVG PFMSEKSSPRICVSAVPMATLGPQSLPPWWRAS SPWL*KQTAS*PGGTSSTC**RHPGRPT*KRAT GK*TARVIKLAISMDLVWWTQKLSLWRQRSGQQC HRSTCVWPPRTRDPGASP*CRCCGLRP*PAPAR STRTSGWSTWSTWWFAPPSHTHAEETSRSTWFL PREPSLNFWQRGCWIFPMKGLQTGNS*LSTAGEK RLKGSGPWKSKICHPRSATRRSKGS*KNGASYC MAQQSTRTTPSVPISPARGCWSSQPQSWSHPRL PCHPPRWKFLKMRKITQVCAIRSVVTKAVMAPMQ TSA*TASTSAWGVSRPAGSA*VCAPWATLGTQQ QDAVAGATRGVRPAPAELRRSACLAAAGSITTR R*TPV*PSVLQDFMLMKVRKIALNATQAVKSAWM NLRNVLSVKKDSALHGAAAFLTVSQAPTLTQS* SDVGNAITPAEPAWGQAEKSAFTVRKTSTSTTG SVCQPVVRASTQKRCRACPTKCVEGTVLLAGNGR QLCPAKECLEGRVWQHLPQGPGRACCITPLWTG AHLQSC*GAFVPLFTGCIFVGSLLWADTVQQRD KMGSRIARCSHPIGGLRGTSLPMSRSACILCTVD NQRNHVLG*K

Frame 2

AGRGRLWGGTASREPQKSAGKFPRWGAFRASRT ALGPGARSQERGPGGGNAPRPPPPPRLPPAAVL AAAVAAAAALPRRGAALKGGTPPPGALAARAPR EPVAAMPPRAPPAPGPRPPPRAAAATDTAAGAGG AGGAGGAGGPGFRPLAPRPWRWLLLLALPAACS APAAPRLHQPLGGASAGRPGRGGPRGGGARVPQ LGPDWKPGRLLPFLSQQNL*KINLE*QRPSHLPQ NGPPGEMAPATGSETKGEETGAK*PAGPLLQRP HLVQHVVPALWRQEQSLPVGNECPGSVEEGLHR KKRGGHHP**WHREKSP*PGPKL*FLRQLRRERQ *L*PISTI*CQQ*K*TRHSLCGRSCCFSKQFLL HRGHSVQCQNRRHPHAGRRCHRCGRGKVAGHQT QLHRHLQCQLGAGRRRQDGGRARPTG*AGFRVWH *KGPAGPGLHFRLGIWEWRERGGLLLVRWLHQQ HLHHLRQQRHREWLQALVPGRVCLHPGHHLQQW GLL*AKNRHHGSASALYRWPHWDLSLCPHGGGHH RLGSRSKQPVNLEGRPAPASEDIPAGPPESERL ESERRGS*S*PFLWIWFGGRRSSRCGGKEVDSS AIAAHVCGRLGQETQEHPLSAGAADYGPDQRLRG ALGPAGGLLGARGGSHLHLTPTPRRPPDLPGFS LGNQVSTSGKEVAGSFQ*RVYKLGIHDCPLLGR KG*RAVDLGNPRSAIPGPQPGEAREVERMEPHTV WHSRAPVPHLQCPSVPLADAGALSPRAGATQGC PVTLPGGSS*R*GRLHRCVPSGVW*QRL*WPQC RPVLELRPLQPGECQDQQEVRECVPLGLLWGHSS KTLSPVPQGV*DLLQQSCDAVPVLPPRVLSPPG DEHLCDPLSCRILC**KSEKLP*MPPKL*KVRG *T*EMYCL*RRIQPCTGQLHS*L*ARHLL*LRAD QMWGMPSHLRNLRGARQRRVHSLCEKLPLPRLE VCASLW*GLLPRRDAGLAPQSVSKVRSSWRGTA GSCVQQRSAWRAESGSIFPRGRGGHVASPHCGQE PIYRAAEGPSSLCSLDAYLLGPCCGQTPCSSGI RWEAE*QGAHTPLVDYAGRPCPCRAQHASFALS IIRETMFWVK

Sequence PCSK6.bAug05

TCGCGGGCCGAGGACGCCTCTGGGGCGGCACCGCGTCCCGAGAGCCCCAGAAGTCGGCGGGGAAGTTCCCCCGGTGGGGGGCGTTTCGGGCCTCCCGGAC GGCTCTCGGCCCCGGAGCCCGGTCGCAGGAGCGCGGGCCCGGGGGCGGGAACGCGCCGCGGCCGCCTCCTCCTCCCCGGCTCCCGCCCGCGGCGGTGTTG GCGGCGGCGGTGGCGGCGGCGGCGGCGCTTCCCCGGCGCGGAGCGGCTTTAAAAGGCGGCACTCCACCCCCCGGCGCACTCGCAGCTCGGGCGCCGCGCG AGCCTGTCGCCGCTATGCCTCCGCGCGCGCCGCCTGCGCCCGGGCCCCGGCCGCCGCCCCGGGCCGCCGCCGCCACCGACACCGCCGCGGGCGCGGGGGG CGCGGGGGGCGCGGGGGGCGCCGGCGGGCCCGGGTTCCGGCCGCTCGCGCCGCGTCCCTGGCGCTGGCTGCTGCTGCTGGCGCTGCCTGCCGCCTGCTCC GCCCCCGCCGCGCCCCGTCTACACCAACCACTGGGCGGTGCAAGTGCTGGGCGGCCCGGCCGAGGCGGACCGCGTGGCGGCGGCGCACGGGTACCTCAAC TTGGGCCAGATTGGAAACCTGGAAGATTACTACCATTTTTATCACAGCAAAACCTTTAAAAGATCAACCTTGAGTAGCAGAGGCCCTCACACCTTCCTCA GAATGGACCCCCAGGTGAAATGGCTCCAGCAACAGGAAGTGAAACGAAGGGTGAAGAGACAGGTGCGAAGTGACCCGCAGGCCCTTTACTTCAACGACCC CATTTGGTCCAACATGTGGTACCTGCATTGTGGCGACAAGAACAGTCGCTGCCGGTCGGAAATGAATGTCCAGGCAGCGTGGAAGAGGGGCTACACAGGA AAAAACGTGGTGGTCACCATCCTTGATGATGGCATAGAGAGAAATCACCCTGACCTGGCCCCAAATTATGATTCCTACGCCAGCTACGACGTGAACGGCA ATGATTATGACCCATCTCCACGATATGATGCCAGCAATGAAAATAAACACGGCACTCGTTGTGCGGGAGAAGTTGCTGCTTCAGCAAACAATTCCTACTG CATCGTGGGCATAGCGTACAATGCCAAAATAGGAGGCATCCGCATGCTGGACGGCGATGTCACAGATGTGGTCGAGGCAAAGTCGCTGGGCATCAGACCC AACTACATCGACATTTACAGTGCCAGCTGGGGGCCGGACGACGACGGCAAGACGGTGGACGGGCCCGGCCGACTGGCTAAGCAGGCTTTCGAGTATGGCA TTAAAAAGGGCCGGCAGGGCCTGGGCTCCATTTTCGTCTGGGCATCTGGGAATGGCGGGAGAGAGGGGGACTACTGCTCGTGCGATGGCTACACCAACAG CATCTACACCATCTCCGTCAGCAGCGCCACCGAGAATGGCTACAAGCCCTGGTACCTGGAAGAGTGTGCCTCCACCCTGGCCACCACCTACAGCAGTGGG GCCTTTTATGAGCGAAAAATCGTCACCACGGATCTGCGTCAGCGCTGTACCGATGGCCACACTGGGACCTCAGTCTCTGCCCCCATGGTGGCGGGCATCA TCGCCTTGGCTCTAGAAGCAAACAGCCAGTTAACCTGGAGGGACGTCCAGCACCTGCTAGTGAAGACATCCCGGCCGGCCCACCTGAAAGCGAGCGACTG GAAAGTGAACGGCGCGGGTCATAAAGTTAGCCATTTCTATGGATTTGGTTTGGTGGACGCAGAAGCTCTCGTTGTGGAGGCAAAGAAGTGGACAGCAGTG CCATCGCAGCACATGTGTGTGGCCGCCTCGGACAAGAGACCCAGGAGCATCCCCTTAGTGCAGGTGCTGCGGACTACGGCCCTGACCAGCGCCTGCGCGG AGCACTCGGACCAGCGGGTGGTCTACTTGGAGCACGTGGTGGTTCGCACCTCCATCTCACACCCACGCCGAGGAGACCTCCAGATCTACCTGGTTTCTCC CTCGGGAACCAAGTCTCAACTTCTGGCAAAGAGGTTGCTGGATCTTTCCAATGAAGGGTTTACAAACTGGGAATTCATGACTGTCCACTGCTGGGGAGAA AAGGCTGAAGGGCAGTGGACCTTGGAAATCCAAGATCTGCCATCCCAGGTCCGCAACCCGGAGAAGCAAGGGAAGTTGAAAGAATGGAGCCTCATACTGT ATGGCACAGCAGAGCACCCGTACCACACCTTCAGTGCCCATCAGTCCCGCTCGCGGATGCTGGAGCTCTCAGCCCCAGAGCTGGAGCCACCCAAGGCTGC CCTGTCACCCTCCCAGGTGGAAGTTCCTGAAGATGAGGAAGATTACACAGCTCAATCCACCCCAGGCTCTGCTAATATTTTACAGACCAGTGTGTGCCAT CCGGAGTGTGGTGACAAAGGCTGTGATGGCCCCAATGCAGACCAGTGCTTGAACTGCGTCCACTTCAGCCTGGGGAGTGTCAAGACCAGCAGGAAGTGCG TGAGTGTGTGCCCCTTGGGCTACTTTGGGGACACAGCAGCAAGACGCTGTCGCCGGTGCCACAAGGGGTGTGAGACCTGCTCCAGCAGAGCTGCGACGCA GTGCCTGTCTTGCCGCCGCGGGTTCTATCACCACCAGGAGATGAACACCTGTGTGACCCTCTGTCCTGCAGGATTTTATGCTGATGAAAGTCAGAAAAAT TGCCTTAAATGCCACCCAAGCTGTAAAAAGTGCGTGGATGAACCTGAGAAATGTACTGTCTGTAAAGAAGGATTCAGCCTTGCACGGGGCAGCTGCATTC CTGACTGTGAGCCAGGCACCTACTTTGACTCAGAGCTGATCAGATGTGGGGAATGCCATCACACCTGCGGAACCTGCGTGGGGCCAGGCAGAGAAGAGTG CATTCACTGTGCGAAAAACTTCCACTTCCACGACTGGAAGTGTGTGCCAGCCTGTGGTGAGGGCTTCTACCCAGAAGAGATGCCGGGCTTGCCCCACAAA GTGTGTCGAAGGTGTGACGAGAACTGCTTGAGCTGTGCAGGCTCCAGCAGGAACTGTAGCAGGTGTAAGACGGGCTTCACACAGCTGGGGACCTCCTGCA TCACCAACCACACGTGCAGCAACGCTGACGAGACATTCTGCGAGATGGTGAAGTCCAACCGGCTGTGCGAACGGAAGCTCTTCATTCAGTTCTGCTGCCG CACGTGCCTCCTGGCCGGGTAAGGGTGCCTAGCTGCCCACAGAGGGCAGGCACTCCCATCCATCCATCCGTCCACCTTCCTCCAGACTGTCGGCCAGAGT CTGTTTCAGGAGCGGCGCCCTGCACCTGACAGCTTTATCTCCCCAGGAGCAGCATCTCTGAGCACCCAAGCCAGGTGGGTGGTGGCTCTTAAGGAGGTGT TCCTAAAATGGTGATATCCTCTCAAATGCTGCTTGTTGGCTCCAGTCTTCCGACAAACTAACAGGAACAAAATGAATTCTGGGAATCCACAGCTCTGGCT TTGGAGCAGCTTCTGGGACCATAAGTTTACTGAATCTTCAAGACCAAAGCAGAAAAGAAAGGCGCTTGGCATCACACATCACTCTTCTCCCCGTGCTTTT CTGCGGCTGTGTAGTAAATCTCCCCGGCCCAGCTGGCGAACCCTGGGCCATCCTCACATGTGACAAAGGGCCAGCAGTCTACCTGCTCGTTGCCTGCCAC TGAGCAGTCTGGGGACGGTTTGGTCAGACTATAAATAAGATAGGTTTGAGGGCATAAAATGTATGACCACTGGGGCCGGAGTATCTATTTCTACATAGTC AGCTACTTCTGAAACTGCAGCAGTGGCTTAGAAAGTCCAATTCCAAAGCCAGACCAGAAGATTCTATCCCCCGCAGCGCTCTCCTTTGAGCAAGCCGAGC TCTCCTTGTTACCGTGTTCTGTCTGTGTCTTCAGGAGTCTCATGGCCTGAACGACCACCTCGACCTGATGCAGAGCCTTCTGAGGAGAGGCAACAGGAGG CATTCTGTGGCCAGCCAAAAGGTACCCCGATGGCCAAGCAATTCCTCTGAACAAAATGTAAAGCCAGCCATGCATTGTTAATCATCCATCACTTCCCATT TTATGGAATTGCTTTTAAAATACATTTGGCCTCTGCCCTTCAGAAGACTCGTTTTTAAGGTGGAAACTCCTGTGTCTGTGTATATTACAAGCCTACATGA CACAGTTGGATTTATTCTGCCAAACCTGTGTAGGCATTTTATAAGCTACATGTTCTAATTTTTACCGATGTTAATTATTTTGACAAATATTTCATATATT TTCATTGAAATGCACAGATCTGCTTGATCAATTCCCTTGAATAGGGAAGTAACATTTGCCTTAAATTTTTTCGACCTCGTCTTTCTCCATATTGTCCTGC TCCCCTGTTTGACGACAGTGCATTTGCCTTGTCACCTGTGAGCTGGAGAGAACCCAGATGTTGTTTATTGAATCTACAACTCTGAAAGAGAAATCAATGA AGCAAGTACAATGTTAACCCTAAATTAATAAAAGAGTTAACATCCCATGGCAC

PCSK6.F1 (forward primer)
PCSK6.R33 (reverse primer)
Single exon skipping

Frame 0

SRAEDASGAAPRPESPRSRRGSSPGGGRFGPPG RLSAPEPGRRSAGPGAGTRRGRLLLPGSRPRRCW RRRWRRRRRFPGAERL*KAALHPPAHSQLGRRA SLSPLCLRARRLRPGPGRRPGPPPPPTPPRARG ARGARGAPAGPGSGRSRRVPGAGCCCWRCLPPAP PPPRPVYTNHWAVQVLGGPAEADRVAAAHGYLN LGQIGNLEDYYHFYHSKTFKRSTLSSRGPHTFL RMDPQVKWLQQQEVKRRVKRQVRSDPQALYFNDP IWSNMWYLHCGDKNSRCRSEMNVQAAWKRGYTG KNVVVTILDDGIERNHPDLAPNYDSYASYDVNG NDYDPSPRYDASNENKHGTRCAGEVAASANNSYC IVGIAYNAKIGGIRMLDGDVTDVVEAKSLGIRP NYIDIYSASWGPDDDGKTVDGPGRLAKQAFEYG IKKGRQGLGSIFVWASGNGGREGDYCSCDGYTNS IYTISVSSATENGYKPWYLEECASTLATTYSSG AFYERKIVTTDLRQRCTDGHTGTSVSAPMVAGI IALALEANSQLTWRDVQHLLVKTSRPAHLKASDW KVNGAGHKVSHFYGFGLVDAEALVVEAKKWTAV PSQHMCVAASDKRPRSIPLVQVLRTTALTSACA EHSDQRVVYLEHVVVRTSISHPRRGDLQIYLVSP SGTKSQLLAKRLLDLSNEGFTNWEFMTVHCWGE KAEGQWTLEIQDLPSQVRNPEKQGKLKEWSLIL YGTAEHPYHTFSAHQSRSRMLELSAPELEPPKAA LSPSQVEVPEDEEDYTAQSTPGSANILQTSVCH PECGDKGCDGPNADQCLNCVHFSLGSVKTSRKC VSVCPLGYFGDTAARRCRRCHKGCETCSSRAATQ CLSCRRGFYHHQEMNTCVTLCPAGFYADESQKN CLKCHPSCKKCVDEPEKCTVCKEGFSLARGSCI PDCEPGTYFDSELIRCGECHHTCGTCVGPGREEC IHCAKNFHFHDWKCVPACGEGFYPEEMPGLPHK VCRRCDENCLSCAGSSRNCSRCKTGFTQLGTSC ITNHTCSNADETFCEMVKSNRLCERKLFIQFCCR TCLLAG*GCLAAHRGQALPSIHPSTFLQTVGQS LFQERRPAPDSFISPGAASLSTQARWVVALKEV FLKW*YPLKCCLLAPVFRQTNRNKMNSGNPQLWL WSSFWDHKFTESSRPKQKRKALGITHHSSPRAF LRLCSKSPRPSWRTLGHPHM*QRASSLPARCLP LSSLGTVWSDYK*DRFEGIKCMTTGAGVSIST*S ATSETAAVA*KVQFQSQTRRFYPPQRSPLSKPS SPCYRVLSVSSGVSWPERPPRPDAEPSEERQQE AFCGQPKGTPMAKQFL*TKCKASHALLIIHHFPF YGIAFKIHLASALQKTRF*GGNSCVCVYYKPT* HSWIYSAKPV*AFYKLHVLIFTDVNYFDKYFIY FH*NAQICLINSLE*GSNICLKFFRPRLSPYCPA PLFDDSAFALSPVSWREPRCCLLNLQL*KRNQ* SKYNVNPKLIKELTSHG

Frame 1

RGPRTPLGRHRVPRAPEVGGEVPPVGGVSGLPD GSRPRSPVAGARARGRERAAAASSSPAPARGGV GGGGGGGGGASPARSGFKRRHSTPRRTRSSGAAR ACRRYASARAACARAPAAAPGRRRHRHRRGRGG RGGRGGRRRARVPAARAASLALAAAAGAACRLL RPRRAPSTPTTGRCKCWAARPRRTAWRRRTGTST WARLETWKITTIFITAKPLKDQP*VAEALTPSS EWTPR*NGSSNRK*NEG*RDRCEVTRRPFTSTT PFGPTCGTCIVATRTVAAGRK*MSRQRGRGATQE KTWWSPSLMMA*REITLTWPQIMIPTPATT*TA MIMTHLHDMMPAMKINTALVVREKLLLQQTIPT ASWA*RTMPK*EASACWTAMSQMWSRQSRWASDP TTSTFTVPAGGRTTTARRWTGPADWLSRLSSMA LKRAGRAWAPFSSGHLGMAGERGTTARAMATPT ASTPSPSAAPPRMATSPGTWKSVPPPWPPPTAVG PFMSEKSSPRICVSAVPMATLGPQSLPPWWRAS SPWL*KQTAS*PGGTSSTC**RHPGRPT*KRAT GK*TARVIKLAISMDLVWWTQKLSLWRQRSGQQC HRSTCVWPPRTRDPGASP*CRCCGLRP*PAPAR STRTSGWSTWSTWWFAPPSHTHAEETSRSTWFL PREPSLNFWQRGCWIFPMKGLQTGNS*LSTAGEK RLKGSGPWKSKICHPRSATRRSKGS*KNGASYC MAQQSTRTTPSVPISPARGCWSSQPQSWSHPRL PCHPPRWKFLKMRKITQLNPPQALLIFYRPVCAI RSVVTKAVMAPMQTSA*TASTSAWGVSRPAGSA *VCAPWATLGTQQQDAVAGATRGVRPAPAELRR SACLAAAGSITTRR*TPV*PSVLQDFMLMKVRKI ALNATQAVKSAWMNLRNVLSVKKDSALHGAAAF LTVSQAPTLTQS*SDVGNAITPAEPAWGQAEKS AFTVRKTSTSTTGSVCQPVVRASTQKRCRACPTK CVEGVTRTA*AVQAPAGTVAGVRRASHSWGPPA SPTTRAATLTRHSARW*SPTGCANGSSSFSSAA ARASWPGKGA*LPTEGRHSHPSIRPPSSRLSARV CFRSGALHLTALSPQEQHL*APKPGGWWLLRRC S*NGDILSNAACWLQSSDKLTGTK*ILGIHSSG FGAASGTISLLNLQDQSRKERRLASHITLLPVLF CGCVVNLPGPAGEPWAILTCDKGPAVYLLVACH *AVWGRFGQTINKIGLRA*NV*PLGPEYLFLHS QLLLKLQQWLRKSNSKARPEDSIPRSALL*ASRA LLVTVFCLCLQESHGLNDHLDLMQSLLRRGNRR HSVASQKVPRWPSNSSEQNVKPAMHC*SSITSH FMELLLKYIWPLPFRRLVFKVETPVSVYITSLHD TVGFILPNLCRHFISYMF*FLPMLIILTNISYI FIEMHRSA*SIPLNREVTFALNFFDLVFLHIVL LPCLTTVHLPCHL*AGENPDVVY*IYNSEREINE ASTMLTLN**KS*HPMA

Frame 2

AGRGRLWGGTASREPQKSAGKFPRWGAFRASRT ALGPGARSQERGPGGGNAPRPPPPPRLPPAAVL AAAVAAAAALPRRGAALKGGTPPPGALAARAPR EPVAAMPPRAPPAPGPRPPPRAAAATDTAAGAGG AGGAGGAGGPGFRPLAPRPWRWLLLLALPAACS APAAPRLHQPLGGASAGRPGRGGPRGGGARVPQ LGPDWKPGRLLPFLSQQNL*KINLE*QRPSHLPQ NGPPGEMAPATGSETKGEETGAK*PAGPLLQRP HLVQHVVPALWRQEQSLPVGNECPGSVEEGLHR KKRGGHHP**WHREKSP*PGPKL*FLRQLRRERQ *L*PISTI*CQQ*K*TRHSLCGRSCCFSKQFLL HRGHSVQCQNRRHPHAGRRCHRCGRGKVAGHQT QLHRHLQCQLGAGRRRQDGGRARPTG*AGFRVWH *KGPAGPGLHFRLGIWEWRERGGLLLVRWLHQQ HLHHLRQQRHREWLQALVPGRVCLHPGHHLQQW GLL*AKNRHHGSASALYRWPHWDLSLCPHGGGHH RLGSRSKQPVNLEGRPAPASEDIPAGPPESERL ESERRGS*S*PFLWIWFGGRRSSRCGGKEVDSS AIAAHVCGRLGQETQEHPLSAGAADYGPDQRLRG ALGPAGGLLGARGGSHLHLTPTPRRPPDLPGFS LGNQVSTSGKEVAGSFQ*RVYKLGIHDCPLLGR KG*RAVDLGNPRSAIPGPQPGEAREVERMEPHTV WHSRAPVPHLQCPSVPLADAGALSPRAGATQGC PVTLPGGSS*R*GRLHSSIHPRLC*YFTDQCVP SGVW*QRL*WPQCRPVLELRPLQPGECQDQQEVR ECVPLGLLWGHSSKTLSPVPQGV*DLLQQSCDA VPVLPPRVLSPPGDEHLCDPLSCRILC**KSEK LP*MPPKL*KVRG*T*EMYCL*RRIQPCTGQLHS *L*ARHLL*LRADQMWGMPSHLRNLRGARQRRV HSLCEKLPLPRLEVCASLW*GLLPRRDAGLAPQ SVSKV*RELLELCRLQQEL*QV*DGLHTAGDLLH HQPHVQQR*RDILRDGEVQPAVRTEALHSVLLP HVPPGRVRVPSCPQRAGTPIHPSVHLPPDCRPE SVSGAAPCT*QLYLPRSSISEHPSQVGGGS*GGV PKMVISSQMLLVGSSLPTN*QEQNEFWESTALA LEQLLGP*VY*IFKTKAEKKGAWHHTSLFSPCF SAAV**ISPAQLANPGPSSHVTKGQQSTCSLPAT EQSGDGLVRL*IR*V*GHKMYDHWGRSIYFYIV SYF*NCSSGLESPIPKPDQKILSPAALSFEQAE LSLLPCSVCVFRSLMA*TTTST*CRAF*GEATGG ILWPAKRYPDGQAIPLNKM*SQPCIVNHPSLPI LWNCF*NTFGLCPSEDSFLRWKLLCLCILQAYM TQLDLFCQTCVGIL*ATCSNFYRC*LF*QIFHIF SLKCTDLLDQFP*IGK*HLP*IFSTSSFSILSC SPV*RQCICLVTCELERTQMLFIESTTLKEKSM KQVQC*P*INKRVNIPWH

Sequence PCSK6.aAug05

TCGCGGGCCGAGGACGCCTCTGGGGCGGCACCGCGTCCCGAGAGCCCCAGAAGTCGGCGGGGAAGTTCCCCCGGTGGGGGGCGTTTCGGGCCTCCCGGAC GGCTCTCGGCCCCGGAGCCCGGTCGCAGGAGCGCGGGCCCGGGGGCGGGAACGCGCCGCGGCCGCCTCCTCCTCCCCGGCTCCCGCCCGCGGCGGTGTTG GCGGCGGCGGTGGCGGCGGCGGCGGCGCTTCCCCGGCGCGGAGCGGCTTTAAAAGGCGGCACTCCACCCCCCGGCGCACTCGCAGCTCGGGCGCCGCGCG AGCCTGTCGCCGCTATGCCTCCGCGCGCGCCGCCTGCGCCCGGGCCCCGGCCGCCGCCCCGGGCCGCCGCCGCCACCGACACCGCCGCGGGCGCGGGGGG CGCGGGGGGCGCGGGGGGCGCCGGCGGGCCCGGGTTCCGGCCGCTCGCGCCGCGTCCCTGGCGCTGGCTGCTGCTGCTGGCGCTGCCTGCCGCCTGCTCC GCCCCCGCCGCGCCCCGTCTACACCAACCACTGGGCGGTGCAAGTGCTGGGCGGCCCGGCCGAGGCGGACCGCGTGGCGGCGGCGCACGGGTACCTCAAC TTGGGCCAGATTGGAAACCTGGAAGATTACTACCATTTTTATCACAGCAAAACCTTTAAAAGATCAACCTTGAGTAGCAGAGGCCCTCACACCTTCCTCA GAATGGACCCCCAGGTGAAATGGCTCCAGCAACAGGAAGTGAAACGAAGGGTGAAGAGACAGGTGCGAAGTGACCCGCAGGCCCTTTACTTCAACGACCC CATTTGGTCCAACATGTGGTACCTGCATTGTGGCGACAAGAACAGTCGCTGCCGGTCGGAAATGAATGTCCAGGCAGCGTGGAAGAGGGGCTACACAGGA AAAAACGTGGTGGTCACCATCCTTGATGATGGCATAGAGAGAAATCACCCTGACCTGGCCCCAAATTATGATTCCTACGCCAGCTACGACGTGAACGGCA ATGATTATGACCCATCTCCACGATATGATGCCAGCAATGAAAATAAACACGGCACTCGTTGTGCGGGAGAAGTTGCTGCTTCAGCAAACAATTCCTACTG CATCGTGGGCATAGCGTACAATGCCAAAATAGGAGGCATCCGCATGCTGGACGGCGATGTCACAGATGTGGTCGAGGCAAAGTCGCTGGGCATCAGACCC AACTACATCGACATTTACAGTGCCAGCTGGGGGCCGGACGACGACGGCAAGACGGTGGACGGGCCCGGCCGACTGGCTAAGCAGGCTTTCGAGTATGGCA TTAAAAAGGGCCGGCAGGGCCTGGGCTCCATTTTCGTCTGGGCATCTGGGAATGGCGGGAGAGAGGGGGACTACTGCTCGTGCGATGGCTACACCAACAG CATCTACACCATCTCCGTCAGCAGCGCCACCGAGAATGGCTACAAGCCCTGGTACCTGGAAGAGTGTGCCTCCACCCTGGCCACCACCTACAGCAGTGGG GCCTTTTATGAGCGAAAAATCGTCACCACGGATCTGCGTCAGCGCTGTACCGATGGCCACACTGGGACCTCAGTCTCTGCCCCCATGGTGGCGGGCATCA TCGCCTTGGCTCTAGAAGCAAACAGCCAGTTAACCTGGAGGGACGTCCAGCACCTGCTAGTGAAGACATCCCGGCCGGCCCACCTGAAAGCGAGCGACTG GAAAGTGAACGGCGCGGGTCATAAAGTTAGCCATTTCTATGGATTTGGTTTGGTGGACGCAGAAGCTCTCGTTGTGGAGGCAAAGAAGTGGACAGCAGTG CCATCGCAGCACATGTGTGTGGCCGCCTCGGACAAGAGACCCAGGAGCATCCCCTTAGTGCAGGTGCTGCGGACTACGGCCCTGACCAGCGCCTGCGCGG AGCACTCGGACCAGCGGGTGGTCTACTTGGAGCACGTGGTGGTTCGCACCTCCATCTCACACCCACGCCGAGGAGACCTCCAGATCTACCTGGTTTCTCC CTCGGGAACCAAGTCTCAACTTCTGGCAAAGAGGTTGCTGGATCTTTCCAATGAAGGGTTTACAAACTGGGAATTCATGACTGTCCACTGCTGGGGAGAA AAGGCTGAAGGGCAGTGGACCTTGGAAATCCAAGATCTGCCATCCCAGGTCCGCAACCCGGAGAAGCAAGGGAAGTTGAAAGAATGGAGCCTCATACTGT ATGGCACAGCAGAGCACCCGTACCACACCTTCAGTGCCCATCAGTCCCGCTCGCGGATGCTGGAGCTCTCAGCCCCAGAGCTGGAGCCACCCAAGGCTGC CCTGTCACCCTCCCAGGTGGAAGTTCCTGAAGATGAGGAAGATTACACAGCTCAATCCACCCCAGGCTCTGCTAATATTTTACAGACCAGTGTGTGCCAT CCGGAGTGTGGTGACAAAGGCTGTGATGGCCCCAATGCAGACCAGTGCTTGAACTGCGTCCACTTCAGCCTGGGGAGTGTCAAGACCAGCAGGAAGTGCG TGAGTGTGTGCCCCTTGGGCTACTTTGGGGACACAGCAGCAAGACGCTGTCGCCGGTGCCACAAGGGGTGTGAGACCTGCTCCAGCAGAGCTGCGACGCA GTGCCTGTCTTGCCGCCGCGGGTTCTATCACCACCAGGAGATGAACACCTGTGTGACCCTCTGTCCTGCAGGATTTTATGCTGATGAAAGTCAGAAAAAT TGCCTTAAATGCCACCCAAGCTGTAAAAAGTGCGTGGATGAACCTGAGAAATGTACTGTCTGTAAAGAAGGATTCAGCCTTGCACGGGGCAGCTGCATTC CTGACTGTGAGCCAGGCACCTACTTTGACTCAGAGCTGATCAGATGTGGGGAATGCCATCACACCTGCGGAACCTGCGTGGGGCCAGGCAGAGAAGAGTG CATTCACTGTGCGAAAAACTTCCACTTCCACGACTGGAAGTGTGTGCCAGCCTGTGGTGAGGGCTTCTACCCAGAAGAGATGCCGGGCTTGCCCCACAAA GTGTGTCGAAGGTACGGTCCTCCTGGCGGGGAACGGCAGGCAGCTGTGTCCAGCAAAGGAGTGCCTGGAGGGCAGAGTCTGGCAGCATCTTCCCCAGGGG CCGGGGAGGGCATGTTGCATCACCCCACTGTGGACAGGAGCCCATTTACAGAGCTGCTGAGGGGCCTTCGTCCCTTTGTTCACTGGATGCATATTTGTTG GGTCCCTGCTGTGGGCAGACACCGTGCAGCAGCGGGATAAGATGGGAAGCAGAATAGCAAGGTGCTCACACCCCATTGGTGGACTACGCGGGACGTCCCT GCCCATGTCGCGCTCAGCATGCATCCTTTGCACTGTCGATAATCAGAGAAACCATGTTTTGGGTTAAAAA

PCSK6.F1 (forward primer)
PCSK6.R33 (reverse primer)
Single exon skipping

Frame 0

SRAEDASGAAPRPESPRSRRGSSPGGGRFGPPG RLSAPEPGRRSAGPGAGTRRGRLLLPGSRPRRCW RRRWRRRRRFPGAERL*KAALHPPAHSQLGRRA SLSPLCLRARRLRPGPGRRPGPPPPPTPPRARG ARGARGAPAGPGSGRSRRVPGAGCCCWRCLPPAP PPPRPVYTNHWAVQVLGGPAEADRVAAAHGYLN LGQIGNLEDYYHFYHSKTFKRSTLSSRGPHTFL RMDPQVKWLQQQEVKRRVKRQVRSDPQALYFNDP IWSNMWYLHCGDKNSRCRSEMNVQAAWKRGYTG KNVVVTILDDGIERNHPDLAPNYDSYASYDVNG NDYDPSPRYDASNENKHGTRCAGEVAASANNSYC IVGIAYNAKIGGIRMLDGDVTDVVEAKSLGIRP NYIDIYSASWGPDDDGKTVDGPGRLAKQAFEYG IKKGRQGLGSIFVWASGNGGREGDYCSCDGYTNS IYTISVSSATENGYKPWYLEECASTLATTYSSG AFYERKIVTTDLRQRCTDGHTGTSVSAPMVAGI IALALEANSQLTWRDVQHLLVKTSRPAHLKASDW KVNGAGHKVSHFYGFGLVDAEALVVEAKKWTAV PSQHMCVAASDKRPRSIPLVQVLRTTALTSACA EHSDQRVVYLEHVVVRTSISHPRRGDLQIYLVSP SGTKSQLLAKRLLDLSNEGFTNWEFMTVHCWGE KAEGQWTLEIQDLPSQVRNPEKQGKLKEWSLIL YGTAEHPYHTFSAHQSRSRMLELSAPELEPPKAA LSPSQVEVPEDEEDYTAQSTPGSANILQTSVCH PECGDKGCDGPNADQCLNCVHFSLGSVKTSRKC VSVCPLGYFGDTAARRCRRCHKGCETCSSRAATQ CLSCRRGFYHHQEMNTCVTLCPAGFYADESQKN CLKCHPSCKKCVDEPEKCTVCKEGFSLARGSCI PDCEPGTYFDSELIRCGECHHTCGTCVGPGREEC IHCAKNFHFHDWKCVPACGEGFYPEEMPGLPHK VCRRYGPPGGERQAAVSSKGVPGGQSLAASSPG AGEGMLHHPTVDRSPFTELLRGLRPFVHWMHICW VPAVGRHRAAAG*DGKQNSKVLTPHWWTTRDVP AHVALSMHPLHCR*SEKPCFGLK

Frame 1

RGPRTPLGRHRVPRAPEVGGEVPPVGGVSGLPD GSRPRSPVAGARARGRERAAAASSSPAPARGGV GGGGGGGGGASPARSGFKRRHSTPRRTRSSGAAR ACRRYASARAACARAPAAAPGRRRHRHRRGRGG RGGRGGRRRARVPAARAASLALAAAAGAACRLL RPRRAPSTPTTGRCKCWAARPRRTAWRRRTGTST WARLETWKITTIFITAKPLKDQP*VAEALTPSS EWTPR*NGSSNRK*NEG*RDRCEVTRRPFTSTT PFGPTCGTCIVATRTVAAGRK*MSRQRGRGATQE KTWWSPSLMMA*REITLTWPQIMIPTPATT*TA MIMTHLHDMMPAMKINTALVVREKLLLQQTIPT ASWA*RTMPK*EASACWTAMSQMWSRQSRWASDP TTSTFTVPAGGRTTTARRWTGPADWLSRLSSMA LKRAGRAWAPFSSGHLGMAGERGTTARAMATPT ASTPSPSAAPPRMATSPGTWKSVPPPWPPPTAVG PFMSEKSSPRICVSAVPMATLGPQSLPPWWRAS SPWL*KQTAS*PGGTSSTC**RHPGRPT*KRAT GK*TARVIKLAISMDLVWWTQKLSLWRQRSGQQC HRSTCVWPPRTRDPGASP*CRCCGLRP*PAPAR STRTSGWSTWSTWWFAPPSHTHAEETSRSTWFL PREPSLNFWQRGCWIFPMKGLQTGNS*LSTAGEK RLKGSGPWKSKICHPRSATRRSKGS*KNGASYC MAQQSTRTTPSVPISPARGCWSSQPQSWSHPRL PCHPPRWKFLKMRKITQLNPPQALLIFYRPVCAI RSVVTKAVMAPMQTSA*TASTSAWGVSRPAGSA *VCAPWATLGTQQQDAVAGATRGVRPAPAELRR SACLAAAGSITTRR*TPV*PSVLQDFMLMKVRKI ALNATQAVKSAWMNLRNVLSVKKDSALHGAAAF LTVSQAPTLTQS*SDVGNAITPAEPAWGQAEKS AFTVRKTSTSTTGSVCQPVVRASTQKRCRACPTK CVEGTVLLAGNGRQLCPAKECLEGRVWQHLPQG PGRACCITPLWTGAHLQSC*GAFVPLFTGCIFV GSLLWADTVQQRDKMGSRIARCSHPIGGLRGTSL PMSRSACILCTVDNQRNHVLG*K

Frame 2

AGRGRLWGGTASREPQKSAGKFPRWGAFRASRT ALGPGARSQERGPGGGNAPRPPPPPRLPPAAVL AAAVAAAAALPRRGAALKGGTPPPGALAARAPR EPVAAMPPRAPPAPGPRPPPRAAAATDTAAGAGG AGGAGGAGGPGFRPLAPRPWRWLLLLALPAACS APAAPRLHQPLGGASAGRPGRGGPRGGGARVPQ LGPDWKPGRLLPFLSQQNL*KINLE*QRPSHLPQ NGPPGEMAPATGSETKGEETGAK*PAGPLLQRP HLVQHVVPALWRQEQSLPVGNECPGSVEEGLHR KKRGGHHP**WHREKSP*PGPKL*FLRQLRRERQ *L*PISTI*CQQ*K*TRHSLCGRSCCFSKQFLL HRGHSVQCQNRRHPHAGRRCHRCGRGKVAGHQT QLHRHLQCQLGAGRRRQDGGRARPTG*AGFRVWH *KGPAGPGLHFRLGIWEWRERGGLLLVRWLHQQ HLHHLRQQRHREWLQALVPGRVCLHPGHHLQQW GLL*AKNRHHGSASALYRWPHWDLSLCPHGGGHH RLGSRSKQPVNLEGRPAPASEDIPAGPPESERL ESERRGS*S*PFLWIWFGGRRSSRCGGKEVDSS AIAAHVCGRLGQETQEHPLSAGAADYGPDQRLRG ALGPAGGLLGARGGSHLHLTPTPRRPPDLPGFS LGNQVSTSGKEVAGSFQ*RVYKLGIHDCPLLGR KG*RAVDLGNPRSAIPGPQPGEAREVERMEPHTV WHSRAPVPHLQCPSVPLADAGALSPRAGATQGC PVTLPGGSS*R*GRLHSSIHPRLC*YFTDQCVP SGVW*QRL*WPQCRPVLELRPLQPGECQDQQEVR ECVPLGLLWGHSSKTLSPVPQGV*DLLQQSCDA VPVLPPRVLSPPGDEHLCDPLSCRILC**KSEK LP*MPPKL*KVRG*T*EMYCL*RRIQPCTGQLHS *L*ARHLL*LRADQMWGMPSHLRNLRGARQRRV HSLCEKLPLPRLEVCASLW*GLLPRRDAGLAPQ SVSKVRSSWRGTAGSCVQQRSAWRAESGSIFPRG RGGHVASPHCGQEPIYRAAEGPSSLCSLDAYLL GPCCGQTPCSSGIRWEAE*QGAHTPLVDYAGRP CPCRAQHASFALSIIRETMFWVK