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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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Bibliografiska uppgifter
Huvudupphovsmän: Sobczak, Krzysztof, Wheeler, Thurman M, Wang, Wenli, Thornton, Charles A
Materialtyp: Artigo
Språk:Inglês
Publicerad: Nature Publishing Group 2013
Ämnen:
Länkar: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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