Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules

Kaalak Reddy; Jana R Jenquin; John D Cleary; J Andrew Berglund

doi:10.3390/ijms20164017

Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules

Int J Mol Sci. 2019 Aug 17;20(16):4017. doi: 10.3390/ijms20164017.

Authors

Kaalak Reddy¹, Jana R Jenquin², John D Cleary³, J Andrew Berglund^{4

5}

Affiliations

¹ The RNA Institute, University at Albany-SUNY, Albany, NY 12222, USA. [email protected].
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. [email protected].
⁵ Center for NeuroGenetics and Biochemistry & Molecular Biology, University of Florida, Gainesville, FL 32608, USA. [email protected].

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.

Publication types

Review

MeSH terms

Animals
Humans
Myotonic Dystrophy / drug therapy*
Myotonic Dystrophy / metabolism
Myotonic Dystrophy / therapy
RNA, Messenger / antagonists & inhibitors*

Substances

RNA, Messenger

Abstract

Publication types

MeSH terms

Substances

Grants and funding