In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.

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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Những tác giả chính:	Aoki, Y, Nakamura, A, Yokota, T, Saito, T, Okazawa, H, Nagata, T, Takeda, S
Định dạng:	Journal article
Ngôn ngữ:	English
Được phát hành:	2010

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