Nuclear RNA foci in the heart in myotonic dystrophy.

Mankodi, Ami; Lin, Xiaoyan; Blaxall, Burns C; Swanson, Maurice S; Thornton, Charles A · Circ Res · 2005

basic_science · Level V

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Abstract

The disease mechanism underlying myotonic dystrophy type 1 (DM1) pathogenesis in skeletal muscle may involve sequestration of RNA binding proteins in nuclear foci of expanded poly(CUG) RNA. Here we report evidence for a parallel mechanism in the heart. Accumulation of expanded poly(CUG) RNA in nuclear foci is associated with sequestration of muscleblind proteins and abnormal regulation of alternative splicing in DM1 cardiac muscle. A toxic effect of RNA with an expanded repeat may contribute to cardiac disease in DM1.

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