Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.

Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.

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Duchenne muscular dystrophy (DMD) is a fatal muscle wasting disorder caused by mutations in the dystrophin gene. Antisense-mediated exon skipping is one of the most promising approaches for the treatment of DMD but still faces personalized medicine challenges as different mutations found in DMD pati...

書誌詳細
主要な著者:	Goyenvalle, A, Wright, J, Babbs, A, Wilkins, V, Garcia, L, Davies, K
フォーマット:	Journal article
言語:	English
出版事項:	2012

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