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A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in a <i>Drosophila</i> Model of Myotonic Dystrophy

1 Jun 2016

Abstract excerpt

Myotonic dystrophy is the most common form of adult-onset muscular dystrophy, originating in a CTG repeat expansion in the DMPK gene. The expanded CUG transcript sequesters MBNL1, a key regulator of alternative splicing, leading to the misregulation of numerous pre-mRNAs. We report an RNA-targeted agent as a possible lead compound for the treatment of myotonic dystrophy type 1 (DM1) that reveals both the promise...

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PMID: 27245480
DOI: 10.1002/cmdc.201600081

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A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in a <i>Drosophila</i> Model of Myotonic DystrophyPMID 27245480Read ↗

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