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Article

Myotonic dystrophy: candidate small molecule therapeutics

2 Aug 2017

Abstract excerpt

Myotonic dystrophy type 1 (DM1) is a rare multisystemic neuromuscular disorder caused by expansion of CTG trinucleotide repeats in the noncoding region of the DMPK gene. Mutant DMPK transcripts are toxic and alter gene expression at several levels. Chiefly, the secondary structure formed by CUGs has a strong propensity to capture and retain proteins, like those of the muscleblind-like (MBNL) family. Sequestered...

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PMID: 28780071
DOI: 10.1016/j.drudis.2017.07.011

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