Article
By‐passing the nonsense mutation in the 4<sup><i>CV</i></sup> mouse model of muscular dystrophy by induced exon skipping
12 Nov 2008
Abstract excerpt
BACKGROUND: Duchenne muscular dystrophy (DMD), a severe neuromuscular disorder, is caused by protein-truncating mutations in the dystrophin gene. Absence of functional dystrophin renders muscle fibres more vulnerable to damage and necrosis. We report antisense oligomer (AO) induced exon skipping in the B6Ros.Cg-Dmd(mdx-4Cv)/J (4(CV)) mouse, a muscular dystrophy model arising from a nonsense mutation in dystrophin...
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