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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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Detalhes bibliográficos
Main Authors: Sobczak, Krzysztof, Wheeler, Thurman M, Wang, Wenli, Thornton, Charles A
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2013
Assuntos:
Acesso em linha: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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