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...

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Hlavní autoři: Goyenvalle, Aurélie, Wright, Jordan, Babbs, Arran, Wilkins, Vivienne, Garcia, Luis, Davies, Kay E
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2012
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3369406/
https://ncbi.nlm.nih.gov/pubmed/22354379
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.26
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