[Myotonic dystrophy: therapeutic approaches to RNA toxicity].

Abstract excerpt

Myotonic dystrophy (DM) is the most common hereditary muscle disease in adults, caused by unstable genomic expansions of simple sequence repeats. The mutant RNA transcripts containing the expanded repeat give rise to a toxic gainoffunction by perturbing splicing factors in the nucleus, leading to the misregulation of alternative pre-mRNA splicing. Although no curative treatment exists, recent advances in research...

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