The emperor's new dystrophin: finding sense in the noise von Wilton, S.D, Veedu, R.N, Fletcher, S

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P.6.6 Splice switching Antisense Oligonucleotides: A “kink” in the design? von Price, L, Mitrpant, C, Fletcher, S, Wilton, S.D

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Co-regulation of survival of motor neuron and Bcl-xL expression: Implications for neuroprotection in spinal muscular atrophy von Anderton, R.S, Price, L.L, Turner, B.J, Meloni, B.P, Mitrpant, C, Mastaglia, F.L, Goh, C, Wilton, S.D, Boulos, S

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P.11.5 PMO-mediated dystrophin exon 23 skipping restores mitochondrial function in the mdx mouse heart von Fletcher, S, Viola, H.M, Adams, A.M, Wilton, S.D, Hool, L.C

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P.20.2 Restoration of dystrophin expression after skipping of single and double exon DMD duplications in patient-derived cell lines using antisense oligonucleotide and AAV-U7snRNA... von Vulin, A, Wein, N, Findlay, A.R, Wilton, S.D, Flanigan, K.M

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Massive Idiosyncratic Exon Skipping Corrects the Nonsense Mutation in Dystrophic Mouse Muscle and Produces Functional Revertant Fibers by Clonal Expansion von Lu, Q. L., Morris, G. E., Wilton, S. D., Ly, T., O. V. Artem'yeva, Strong, P., Partridge, T. A.

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G.P.61 Influence of HLA-DRB3 on susceptibility to sporadic inclusion body myositis von Rojana-udomsart, R, Mitrpant, C, Wilton, S.D, Mastaglia, F.L

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P4.30 Multiple exon skipping strategies to by-pass selected dystrophin mutations von Adkin, C, Meloni, P, Fletcher, S, Wilton, S.D

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O10 Development of PMO antisense oligonucleotides for treatment of Spinal muscular atrophy von Burghes, A.H.M, McGovern, V.L, Porensky, P.N, Arnold, W.D, Mitrpant, C, Price, L, Fletcher, S, Wilton, S.D

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P.6.5 Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing von Mitrpant, C, Porensky, P.N, Zhou, H, Price, L, Muntoni, F, Fletcher, S, Wilton, S.D, Burghes, A.H.M

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G.P.78 Dystrophin isoforms with incomplete β dystroglycan and syntrophin binding domains retain partial function von Fletcher, S, Adams, A.M, Greer, K, Johnsen, R.D, Adkin, C, Wilton, S.D

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T.I.4 Splice manipulation therapies: Opportunities and challenges von Wilton, S.D, Fletcher, S

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Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle von Wells, K.E., Fletcher, S., Mann, C.J., Wilton, S.D., Wells, D.J.

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P3.11 Antisense oligomer mediated retention of SMN2 intron and exon 7 leads to a novel SMN transcript and increased functional SMN protein in Spinal Muscular Atrophy fibroblasts von Mitrpant, C, Price, L, Johnsen, R.D, Burghes, A.H, Fletcher, S, Wilton, S.D

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P18 Transient mouse models for the preclinical evaluation of therapeutic dystrophin exon skipping strategies von Wilton, S.D, Adams, A, Adkin, C, Greer, K, Johnsen, R, Fletcher, S

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D.O.2 Microarray testing for developmental delay reveals an expanded clinical spectrum of dystrophinopathies von Howell, K.B, Forbes, R, Burgess, T, O’Reilly, D, DuSart, D, Chong, B, Clarke, N, McLean, C.A, Leventer, R.J, Laing, N.G, Wilton, S.D, Ryan, M.M

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P3.32 Exon/intron 7 inclusion: an alternative mode of splice intervention in spinal muscular atrophy fibroblasts von Mitrpant, C, Fragall, C.T, Johnsen, R, Burghes, A.H, Fletcher, S, Wilton, S.D

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T.P.1.06 Induced non-productive splicing to study muscle gene expression von Wilton, S.D, Fletcher, S, Adams, A.M, Johnsen, R, Greer, K

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T.P.2.03 Characterisation of a complex dystrophin mutation: Assume nothing when designing exon skipping strategies von Fletcher, S, Davis, M.R, Madden, H.R, Wilton, S.D

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P26 Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping von Goyenvalle, A, Babbs, A, Powell, D, Kole, R, Fletcher, S, Wilton, S.D, Davies, K.E

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