Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene

Duchenne muscular dystrophy is a severe muscle-wasting disease caused by mutations in the dystrophin gene that ablate functional protein expression. Although exonic deletions are the most common Duchenne muscular dystrophy lesion, duplications account for 10–15% of reported disease-causing mutations...

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Bibliografiska uppgifter
Huvudupphovsmän: Greer, Kane L, Lochmüller, Hanns, Flanigan, Kevin, Fletcher, Susan, Wilton, Steve D
Materialtyp: Artigo
Språk:Inglês
Publicerad: Nature Publishing Group 2014
Ämnen:
Original Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC3982197/
https://ncbi.nlm.nih.gov/pubmed/24643206
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2014.8
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