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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...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Sobczak, Krzysztof, Wheeler, Thurman M, Wang, Wenli, Thornton, Charles A
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2013
Schlagworte:
Online Zugang: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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