Congenital muscular dystrophies and the extracellular matrix

Semin Pediatr Neurol. 2006 Jun;13(2):80-9. doi: 10.1016/j.spen.2006.06.003.

Abstract

During the past decade, considerable progress in the field of congenital muscular dystrophies (CMDs) had led to the identification of a growing number of causative genes. This genetic progress has uncovered crucial pathophysiological concepts and has been instrumental in redefining clinical phenotypes. Important new pathogenic mechanisms include the disorders of O-mannosyl-linked glycosylation of alpha-dystroglycan as well as the involvement of a collagen type VI in the pathogenesis of congenital disorders of muscle. Thus, an emerging theme among gene products involved in the pathogenesis of congenital muscular dystrophy is their intimate connection to the extracellular matrix. In this review, we focus on the clinical phenotypes that we are correlating with the novel genetic and biochemical findings encountered within CMD. This correlation will frequently lead to a considerably expanded clinical spectrum associated with a given CMD gene.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Extracellular Matrix / classification
  • Extracellular Matrix / genetics*
  • Extracellular Matrix / metabolism*
  • Humans
  • Muscular Dystrophies / classification
  • Muscular Dystrophies / genetics*
  • Muscular Dystrophies / metabolism*
  • Muscular Dystrophies / pathology