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Int J Mol Sci. 2019 Aug 17;20(16). pii: E4017. doi: 10.3390/ijms20164017.

Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules.

Author information

1
The RNA Institute, University at Albany-SUNY, Albany, NY 12222, USA. kreddy2@albany.edu.
2
Center for NeuroGenetics and Biochemistry & Molecular Biology, University of Florida, Gainesville, FL 32608, USA.
3
The RNA Institute, University at Albany-SUNY, Albany, NY 12222, USA.
4
The RNA Institute, University at Albany-SUNY, Albany, NY 12222, USA. aberglund@albany.edu.
5
Center for NeuroGenetics and Biochemistry & Molecular Biology, University of Florida, Gainesville, FL 32608, USA. aberglund@albany.edu.

Abstract

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).

KEYWORDS:

myotonic dystrophy; small molecules; therapies

PMID:
31426500
PMCID:
PMC6720693
DOI:
10.3390/ijms20164017
[Indexed for MEDLINE]
Free PMC Article

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