Novel myosin-based therapies for congenital cardiac and skeletal myopathies

Julien Bruno Emile Ochala; Yin-Biao Sun

doi:10.1136/jmedgenet-2016-103881

Novel myosin-based therapies for congenital cardiac and skeletal myopathies

Julien Bruno Emile Ochala, Yin-Biao Sun

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Abstract

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.

Original language	English
Number of pages	4
Journal	Journal of Medical Genetics
Early online date	13 Jul 2016
DOIs	https://doi.org/10.1136/jmedgenet-2016-103881
Publication status	E-pub ahead of print - 13 Jul 2016

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10.1136/jmedgenet-2016-103881Licence: CC BY

Novel myosin-based therapies_OCHALA_Accepted15June2016_GOLD VoRFinal published version, 274 KBLicence: CC BY

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abstract = "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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N2 - 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.

AB - 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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Novel myosin-based therapies for congenital cardiac and skeletal myopathies

Abstract

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