Novel myosin-based therapies for congenital cardiac and skeletal myopathies

J Med Genet. 2016 Oct;53(10):651-4. doi: 10.1136/jmedgenet-2016-103881. Epub 2016 Jul 13.

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.

Keywords: Cardiomyopathy; Congenital heart disease; Muscle disease.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cardiomyopathies / congenital
  • Cardiomyopathies / metabolism
  • Cardiomyopathies / therapy*
  • Humans
  • Muscular Diseases / congenital
  • Muscular Diseases / metabolism
  • Muscular Diseases / therapy*
  • Myosins*

Substances

  • Myosins