Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene

Protein-truncating mutations in the dystrophin gene lead to the progressive muscle wasting disorder Duchenne muscular dystrophy, whereas in-frame deletions typically manifest as the milder allelic condition, Becker muscular dystrophy. Antisense oligomer-induced exon skipping can modify dystrophin ge...

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Bibliographic Details
Main Authors:	Fletcher, Sue, Adkin, Carl F., Meloni, Penny, Wong, Brenda, Muntoni, Francesco, Kole, Ryszard, Fragall, Clayton, Greer, Kane, Johnsen, Russell, Wilton, Steve D.
Format:	Artigo
Language:	Inglês
Published:	Nature Publishing Group 2012
Subjects:	Original Article
Online Access:	https://ncbi.nlm.nih.gov/pmc/articles/PMC3499695/ https://ncbi.nlm.nih.gov/pubmed/23344648 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2012.40
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Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene

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