Novel myosin-based therapies for congenital cardiac and skeletal myopathies

The dysfunction in a number of inherited cardiac and skeletal myopathies is primarily due to an altered ability of myofilaments to generate force and motion. Despite this crucial knowledge, there are, currently, no effective therapeutic interventions for these diseases. In this short review, we disc...

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Detalles Bibliográficos
Autores principales: Ochala, Julien, Sun, Yin-Biao
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2016
Materias:
Myopathies
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5099184/
https://www.ncbi.nlm.nih.gov/pubmed/27412953
http://dx.doi.org/10.1136/jmedgenet-2016-103881
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author Ochala, Julien
Sun, Yin-Biao
author_facet Ochala, Julien
Sun, Yin-Biao
author_sort Ochala, Julien
collection PubMed
description The dysfunction in a number of inherited cardiac and skeletal myopathies is primarily due to an altered ability of myofilaments to generate force and motion. Despite this crucial knowledge, there are, currently, no effective therapeutic interventions for these diseases. In this short review, we discuss recent findings giving strong evidence that genetically or pharmacologically modulating one of the myofilament proteins, myosin, could alleviate the muscle pathology. This should constitute a research and clinical priority.
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spelling pubmed-50991842016-11-14 Novel myosin-based therapies for congenital cardiac and skeletal myopathies Ochala, Julien Sun, Yin-Biao J Med Genet Myopathies The dysfunction in a number of inherited cardiac and skeletal myopathies is primarily due to an altered ability of myofilaments to generate force and motion. Despite this crucial knowledge, there are, currently, no effective therapeutic interventions for these diseases. In this short review, we discuss recent findings giving strong evidence that genetically or pharmacologically modulating one of the myofilament proteins, myosin, could alleviate the muscle pathology. This should constitute a research and clinical priority. BMJ Publishing Group 2016-10 2016-07-13 /pmc/articles/PMC5099184/ /pubmed/27412953 http://dx.doi.org/10.1136/jmedgenet-2016-103881 Text en Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/ This is an Open Access article distributed in accordance with the terms of the Creative Commons Attribution (CC BY 4.0) license, which permits others to distribute, remix, adapt and build upon this work, for commercial use, provided the original work is properly cited. See: http://creativecommons.org/licenses/by/4.0/
spellingShingle Myopathies
Ochala, Julien
Sun, Yin-Biao
Novel myosin-based therapies for congenital cardiac and skeletal myopathies
title Novel myosin-based therapies for congenital cardiac and skeletal myopathies
title_full Novel myosin-based therapies for congenital cardiac and skeletal myopathies
title_fullStr Novel myosin-based therapies for congenital cardiac and skeletal myopathies
title_full_unstemmed Novel myosin-based therapies for congenital cardiac and skeletal myopathies
title_short Novel myosin-based therapies for congenital cardiac and skeletal myopathies
title_sort novel myosin-based therapies for congenital cardiac and skeletal myopathies
topic Myopathies
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5099184/
https://www.ncbi.nlm.nih.gov/pubmed/27412953
http://dx.doi.org/10.1136/jmedgenet-2016-103881
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