Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.
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...
| 主要な著者: | , , , , , |
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| フォーマット: | Journal article |
| 言語: | English |
| 出版事項: |
2012
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