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Myopathies
Novel myosin-based therapies for congenital cardiac and skeletal myopathies
  1. Julien Ochala1,
  2. Yin-Biao Sun2
  1. 1Centre of Human and Aerospace Physiological Sciences, King's College London, London, UK
  2. 2Randall Division of Cell and Molecular Biophysics, British Heart Foundation Centre of Research Excellence, King's College London, London, UK
  1. Correspondence to Julien Ochala, Centre of Human & Aerospace Physiological Sciences, Faculty of Life Sciences and Medicine, King's College London, Room 3.3, Shepherd's House, Guy's Campus, London SE1 1UL, UK; julien.ochala{at}kcl.ac.uk

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.

  • Congenital heart disease
  • Cardiomyopathy
  • Muscle disease

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/

https://doi.org/10.1136/jmedgenet-2016-103881

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