In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse

A promising therapeutic approach for Duchenne muscular dystrophy (DMD) is exon skipping using antisense oligonucleotides (AOs). In-frame deletions of the hinge 3 region of the dystrophin protein, which is encoded by exons 50 and 51, are predicted to cause a variety of phenotypes. Here, we performed...

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Detalhes bibliográficos
Main Authors: Aoki, Yoshitsugu, Nakamura, Akinori, Yokota, Toshifumi, Saito, Takashi, Okazawa, Hitoshi, Nagata, Tetsuya, Takeda, Shin'ichi
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2010
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2990521/
https://ncbi.nlm.nih.gov/pubmed/20823833
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.186
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