Article
Induced dystrophin exon skipping in human muscle explants.
Neuromuscular disorders : NMD - 1 Oct 2006
McClorey G, Fall A M, Moulton H M, Iversen P L, Rasko J E, Ryan M, Fletcher S, Wilton S D
Abstract excerpt
Antisense oligonucleotide (AO) manipulation of pre-mRNA splicing of the dystrophin gene is showing promise in overcoming Duchenne muscular dystrophy (DMD)-causing mutations. To date, this approach has been limited to studies using animal models or cultured human muscle cells, and evidence that AOs can induce exon skipping in human muscle has yet to be shown. In this study, we used different AO analogues to induce...
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