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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Autores principales: Fletcher, Sue, Adkin, Carl F., Meloni, Penny, Wong, Brenda, Muntoni, Francesco, Kole, Ryszard, Fragall, Clayton, Greer, Kane, Johnsen, Russell, Wilton, Steve D.
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2012
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Original Article
Acceso en línea: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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