Article
Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations.
Neuromuscular disorders : NMD - 1 Dec 2010
Forrest Sarah, Meloni Penny L, Muntoni Francesco, Kim Jihee, Fletcher Sue, Wilton Steve D
Abstract excerpt
Antisense oligomer induced exon skipping is showing promise as a therapy to reduce the severity of Duchenne muscular dystrophy. To date, the focus has been on excluding single exons flanking frame-shifting deletions in the dystrophin gene. However, a third of all Duchenne muscular dystrophy causi...
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