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Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse

Duchenne muscular dystrophy (DMD) is a severe muscle wasting disease arising from defects in the dystrophin gene, typically nonsense or frameshift mutations, that preclude the synthesis of a functional protein. A milder, allelic version of the disease, Becker muscular dystrophy, generally arises fro...

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Autors principals: Mann, Christopher J., Honeyman, Kaite, Cheng, Andy J., Ly, Tina, Lloyd, Frances, Fletcher, Sue, Morgan, Jennifer E., Partridge, Terry A., Wilton, Stephen D.
Format: Artigo
Idioma:Inglês
Publicat: The National Academy of Sciences 2001
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC14541/
https://ncbi.nlm.nih.gov/pubmed/11120883
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