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Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules
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MicroRNA-Based Therapeutic Perspectives in Myotonic Dystrophy

1
Translational Genomics Group, Incliva Health Research Institute, Burjassot, 46100 Valencia, Spain
2
Interdisciplinary Research Structure for Biotechnology and Biomedicine (Eri Biotecmed), University of Valencia, Burjassot, 46100 Valencia, Spain
*
Authors to whom correspondence should be addressed.
Int. J. Mol. Sci. 2019, 20(22), 5600; https://doi.org/10.3390/ijms20225600 (registering DOI)
Received: 16 September 2019 / Revised: 28 October 2019 / Accepted: 30 October 2019 / Published: 9 November 2019
(This article belongs to the Special Issue Myotonic Dystrophy: From Molecular Pathogenesis to Therapeutics)
Myotonic dystrophy involves two types of chronically debilitating rare neuromuscular diseases: type 1 (DM1) and type 2 (DM2). Both share similarities in molecular cause, clinical signs, and symptoms with DM2 patients usually displaying milder phenotypes. It is well documented that key clinical symptoms in DM are associated with a strong mis-regulation of RNA metabolism observed in patient’s cells. This mis-regulation is triggered by two leading DM-linked events: the sequestration of Muscleblind-like proteins (MBNL) and the mis-regulation of the CUGBP RNA-Binding Protein Elav-Like Family Member 1 (CELF1) that cause significant alterations to their important functions in RNA processing. It has been suggested that DM1 may be treatable through endogenous modulation of the expression of MBNL and CELF1 proteins. In this study, we analyzed the recent identification of the involvement of microRNA (miRNA) molecules in DM and focus on the modulation of these miRNAs to therapeutically restore normal MBNL or CELF1 function. We also discuss additional prospective miRNA targets, the use of miRNAs as disease biomarkers, and additional promising miRNA-based and miRNA-targeting drug development strategies. This review provides a unifying overview of the dispersed data on miRNA available in the context of DM. View Full-Text
Keywords: myotonic dystrophy; microRNA; MBNL proteins; CELF1; miRNA-based drug; miRNA-targeting drug; antisense oligonucleotides; alternative splicing myotonic dystrophy; microRNA; MBNL proteins; CELF1; miRNA-based drug; miRNA-targeting drug; antisense oligonucleotides; alternative splicing
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López Castel, A.; Overby, S.J.; Artero, R. MicroRNA-Based Therapeutic Perspectives in Myotonic Dystrophy. Int. J. Mol. Sci. 2019, 20, 5600.

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