Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS

Myotonic dystrophies are multisystemic diseases characterized not only by muscle and heart dysfunction but also by CNS alteration. They are now recognized as brain diseases affecting newborns and children for myotonic dystrophy type 1 and adults for both myotonic dystrophy type 1 and type 2. In the...

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Autores principales: Gourdon, Genevieve, Meola, Giovanni
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2017
Materias:
Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5397409/
https://www.ncbi.nlm.nih.gov/pubmed/28473756
http://dx.doi.org/10.3389/fncel.2017.00101
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author Gourdon, Genevieve
Meola, Giovanni
author_facet Gourdon, Genevieve
Meola, Giovanni
author_sort Gourdon, Genevieve
collection PubMed
description Myotonic dystrophies are multisystemic diseases characterized not only by muscle and heart dysfunction but also by CNS alteration. They are now recognized as brain diseases affecting newborns and children for myotonic dystrophy type 1 and adults for both myotonic dystrophy type 1 and type 2. In the past two decades, much progress has been made in understanding the mechanisms underlying the DM symptoms allowing development of new molecular therapeutic tools with the ultimate aim of curing the disease. This review describes the state of the art for the characterization of CNS related symptoms, the development of molecular strategies to target the CNS as well as the available tools for screening and testing new possible treatments.
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spelling pubmed-53974092017-05-04 Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS Gourdon, Genevieve Meola, Giovanni Front Cell Neurosci Neuroscience Myotonic dystrophies are multisystemic diseases characterized not only by muscle and heart dysfunction but also by CNS alteration. They are now recognized as brain diseases affecting newborns and children for myotonic dystrophy type 1 and adults for both myotonic dystrophy type 1 and type 2. In the past two decades, much progress has been made in understanding the mechanisms underlying the DM symptoms allowing development of new molecular therapeutic tools with the ultimate aim of curing the disease. This review describes the state of the art for the characterization of CNS related symptoms, the development of molecular strategies to target the CNS as well as the available tools for screening and testing new possible treatments. Frontiers Media S.A. 2017-04-20 /pmc/articles/PMC5397409/ /pubmed/28473756 http://dx.doi.org/10.3389/fncel.2017.00101 Text en Copyright © 2017 Gourdon and Meola. http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Neuroscience
Gourdon, Genevieve
Meola, Giovanni
Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS
title Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS
title_full Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS
title_fullStr Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS
title_full_unstemmed Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS
title_short Myotonic Dystrophies: State of the Art of New Therapeutic Developments for the CNS
title_sort myotonic dystrophies: state of the art of new therapeutic developments for the cns
topic Neuroscience
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5397409/
https://www.ncbi.nlm.nih.gov/pubmed/28473756
http://dx.doi.org/10.3389/fncel.2017.00101
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