Therapeutic Targeting of Signaling Pathways in Muscular Dystrophy

J Mol Med (Berl). 2010 Feb;88(2):155-66. doi: 10.1007/s00109-009-0550-4. Epub 2009 Oct 9.

Abstract

Muscular dystrophy refers to a group of genetic diseases that cause severe muscle weakness and loss of skeletal muscle mass. Although research has helped understanding the molecular basis of muscular dystrophy, there is still no cure for this devastating disorder. Numerous lines of investigation suggest that the primary deficiency of specific proteins causes aberrant activation of several cell signaling pathways in skeletal and cardiac muscle leading to the pathogenesis of muscular dystrophy. Studies using genetic mouse models and pharmacological approaches have provided strong evidence that the modulation of the activity of specific cell signaling pathways has enormous potential to improving the quality of life and extending the life expectancy in muscular dystrophy patients. In this article, we have outlined the current understanding regarding the role of different cell signaling pathways in disease progression with particular reference to different models of muscular dystrophy and the development of therapy.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Calcineurin / metabolism
  • Disease Models, Animal
  • Humans
  • Mice
  • Mitogen-Activated Protein Kinase Kinases / metabolism
  • Muscle, Skeletal / metabolism
  • Muscle, Skeletal / physiopathology
  • Muscular Dystrophies / drug therapy*
  • Muscular Dystrophies / metabolism
  • Muscular Dystrophies / physiopathology*
  • Myocardium / metabolism
  • NF-kappa B / genetics
  • NF-kappa B / metabolism
  • NFATC Transcription Factors / metabolism
  • Signal Transduction / drug effects*

Substances

  • NF-kappa B
  • NFATC Transcription Factors
  • Mitogen-Activated Protein Kinase Kinases
  • Calcineurin