Fukuyama-type congenital muscular dystrophy (FCMD) and alpha-dystroglycanopathy

Congenit Anom (Kyoto). 2003 Jun;43(2):97-104. doi: 10.1111/j.1741-4520.2003.tb01033.x.

Abstract

Fukuyama-type congenital muscular dystrophy (FCMD), Walker-Warburg syndrome (WWS), and muscle-eye-brain (MEB) disease are clinically similar autosomal recessive disorders characterized by congenital muscular dystrophy, lissencephaly, and eye anomalies. Through positional cloning, we identified the gene for FCMD and MEB, which encodes the fukutin protein and the protein O-linked mannose beta1, 2-N-acetylglucosaminy ltransferase (POMGnT1), respectively. Recent studies have revealed that posttranslational modification of alpha-dystroglycan is associated with these congenital muscular dystrophies with brain malformations. In this review Fukuyama-type congenital muscular dystrophy (FCMD), other CMDs with brain malformations, and their relation with alpha-dystroglycan are discussed.

Publication types

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

MeSH terms

  • Animals
  • Brain / pathology
  • Child, Preschool
  • Chromosome Mapping
  • Cytoskeletal Proteins / metabolism*
  • Disease Models, Animal
  • Dystroglycans
  • Female
  • Glycosylation
  • Humans
  • Membrane Glycoproteins / metabolism*
  • Membrane Proteins
  • Mice
  • Muscles / pathology
  • Muscular Dystrophies / genetics*
  • Muscular Dystrophies / metabolism
  • Muscular Dystrophies / physiopathology
  • Proteins / metabolism
  • Transferases

Substances

  • Cytoskeletal Proteins
  • DAG1 protein, human
  • FKTN protein, human
  • Membrane Glycoproteins
  • Membrane Proteins
  • Proteins
  • Dystroglycans
  • Fcmd protein, mouse
  • Transferases