Therapeutics development in myotonic dys
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Erin Pennock Foff; Mani S. Mahadevan
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Article
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2011
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John Wiley and Sons
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English
β 238 KB
## Abstract Myotonic dystrophy (DM1), the most common adult muscular dystrophy, is a multisystem, autosomal dominant genetic disorder caused by an expanded CTG repeat that leads to nuclear retention of a mutant RNA and subsequent RNA toxicity. Significant insights into the molecular mechanisms of R