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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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Hlavní autoři: Sobczak, Krzysztof, Wheeler, Thurman M, Wang, Wenli, Thornton, Charles A
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2013
Témata:
On-line přístup: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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