Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules

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 tar...

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Detalles Bibliográficos
Autores principales: Reddy, Kaalak, Jenquin, Jana R., Cleary, John D., Berglund, J. Andrew
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2019
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6720693/
https://www.ncbi.nlm.nih.gov/pubmed/31426500
http://dx.doi.org/10.3390/ijms20164017
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author Reddy, Kaalak
Jenquin, Jana R.
Cleary, John D.
Berglund, J. Andrew
author_facet Reddy, Kaalak
Jenquin, Jana R.
Cleary, John D.
Berglund, J. Andrew
author_sort Reddy, Kaalak
collection PubMed
description 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).
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spelling pubmed-67206932019-09-10 Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules Reddy, Kaalak Jenquin, Jana R. Cleary, John D. Berglund, J. Andrew Int J Mol Sci Review 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). MDPI 2019-08-17 /pmc/articles/PMC6720693/ /pubmed/31426500 http://dx.doi.org/10.3390/ijms20164017 Text en © 2019 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Reddy, Kaalak
Jenquin, Jana R.
Cleary, John D.
Berglund, J. Andrew
Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules
title Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules
title_full Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules
title_fullStr Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules
title_full_unstemmed Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules
title_short Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules
title_sort mitigating rna toxicity in myotonic dystrophy using small molecules
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6720693/
https://www.ncbi.nlm.nih.gov/pubmed/31426500
http://dx.doi.org/10.3390/ijms20164017
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