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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Autores principales: Greer, Kane L, Lochmüller, Hanns, Flanigan, Kevin, Fletcher, Susan, Wilton, Steve D
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2014
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Original Article
Acceso en línea: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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