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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Goyenvalle, Aurélie, Wright, Jordan, Babbs, Arran, Wilkins, Vivienne, Garcia, Luis, Davies, Kay E
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Nature Publishing Group 2012
Pynciau:
Original Article
Mynediad Ar-lein: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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