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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Aoki, Yoshitsugu, Nakamura, Akinori, Yokota, Toshifumi, Saito, Takashi, Okazawa, Hitoshi, Nagata, Tetsuya, Takeda, Shin'ichi
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Nature Publishing Group 2010
Pynciau:
Original Article
Mynediad Ar-lein: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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