RNA Interference Targeting CUG Repeats in a Mouse Model of Myotonic Dystrophy

Myotonic dystrophy type 1 (DM1) is an RNA dominant disease caused by expression of DM protein kinase (DMPK) transcripts that contain an expanded CUG repeat (CUG(exp)). The toxic mRNA localizes to nuclear foci and sequesters proteins involved in the regulation of alternative splicing, such as, muscle...

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Main Authors: Sobczak, Krzysztof, Wheeler, Thurman M, Wang, Wenli, Thornton, Charles A
Formato: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group 2013
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Original Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3594017/
https://ncbi.nlm.nih.gov/pubmed/23183533
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.222
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