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Article

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

27 Nov 2012

Abstract excerpt

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, muscleblind-like 1 (MBNL1). Here, we used synthetic short interfering RNAs (siRNAs) to target CUG repeats and test the...

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PMID: 23183533
DOI: 10.1038/mt.2012.222

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