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Open AccessReview

Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules

The RNA Institute, University at Albany–SUNY, Albany, NY 12222, USA
Center for NeuroGenetics and Biochemistry & Molecular Biology, University of Florida, Gainesville, FL 32608, USA
Authors to whom correspondence should be addressed.
Int. J. Mol. Sci. 2019, 20(16), 4017;
Received: 2 July 2019 / Revised: 13 August 2019 / Accepted: 16 August 2019 / Published: 17 August 2019
(This article belongs to the Special Issue Myotonic Dystrophy: From Molecular Pathogenesis to Therapeutics)
This review, one in a series on myotonic dystrophy (DM), is focused on the development and potential use of small molecules as therapeutics for DM. The complex mechanisms and pathogenesis of DM are covered in the associated reviews. Here, we examine the various small molecule approaches taken to target the DNA, RNA, and proteins that contribute to disease onset and progression in myotonic dystrophy type 1 (DM1) and 2 (DM2). View Full-Text
Keywords: myotonic dystrophy; small molecules; therapies myotonic dystrophy; small molecules; therapies
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MDPI and ACS Style

Reddy, K.; Jenquin, J.R.; Cleary, J.D.; Berglund, J.A. Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules. Int. J. Mol. Sci. 2019, 20, 4017.

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