Chemical treatment enhances skipping of a mutated exon in the dystrophin gene

Duchenne muscular dystrophy (DMD) is a fatal muscle wasting disease caused by a loss of the dystrophin protein. Control of dystrophin mRNA splicing to convert severe DMD to a milder phenotype is attracting much attention. Here we report a dystrophinopathy patient who has a point mutation in exon 31...

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Detalhes bibliográficos
Main Authors: Nishida, Atsushi, Kataoka, Naoyuki, Takeshima, Yasuhiro, Yagi, Mariko, Awano, Hiroyuki, Ota, Mitsunori, Itoh, Kyoko, Hagiwara, Masatoshi, Matsuo, Masafumi
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2011
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3113229/
https://ncbi.nlm.nih.gov/pubmed/21556062
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ncomms1306
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