Enhanced Exon-skipping Induced by U7 snRNA Carrying a Splicing Silencer Sequence: Promising Tool for DMD Therapy

Duchenne muscular dystrophy (DMD) is a fatal muscle wasting disorder caused by mutations in the dystrophin gene. In most cases, the open-reading frame is disrupted which results in the absence of functional protein. Antisense-mediated exon skipping is one of the most promising approaches for the tre...

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Hlavní autoři: Goyenvalle, Aurélie, Babbs, Arran, van Ommen, Gert-Jan B, Garcia, Luis, Davies, Kay E
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2009
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Original Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835201/
https://ncbi.nlm.nih.gov/pubmed/19455105
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.113
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