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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...
Autores principales: | Nishida, Atsushi, Kataoka, Naoyuki, Takeshima, Yasuhiro, Yagi, Mariko, Awano, Hiroyuki, Ota, Mitsunori, Itoh, Kyoko, Hagiwara, Masatoshi, Matsuo, Masafumi |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2011
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3113229/ https://www.ncbi.nlm.nih.gov/pubmed/21556062 http://dx.doi.org/10.1038/ncomms1306 |
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