Article

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

2000-12-19

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a severe muscle wasting\n disease arising from defects in the dystrophin gene, typically nonsense\n or frameshift mutations, that preclude the synthesis of a functional\n protein. A milder, allelic version of the disease, Becker muscular\n dystrophy, generally arises from in-frame deletions that allow\n synthesis of a shorter but still semifunctional protein. Therapies to\n int...

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Literature Corpus work: 61cf3df2-5b55-5622-8f76-e7a2acd59d5c
DOI: 10.1073/pnas.011408598

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