Limb-girdle muscular dystrophy: diagnostic evaluation, frequency and clues to pathogenesis

Neuromuscul Disord. 2008 Jan;18(1):34-44. doi: 10.1016/j.nmd.2007.08.009. Epub 2007 Sep 25.

Abstract

We characterized the frequency of limb-girdle muscular dystrophy (LGMD) subtypes in a cohort of 76 Australian muscular dystrophy patients using protein and DNA sequence analysis. Calpainopathies (8%) and dysferlinopathies (5%) are the most common causes of LGMD in Australia. In contrast to European populations, cases of LGMD2I (due to mutations in FKRP) are rare in Australasia (3%). We have identified a cohort of patients in whom all common disease candidates have been excluded, providing a valuable resource for identification of new disease genes. Cytoplasmic localization of dysferlin correlates with fiber regeneration in a subset of muscular dystrophy patients. In addition, we have identified a group of patients with unidentified forms of LGMD and with markedly abnormal dysferlin localization that does not correlate with fiber regeneration. This pattern is mimicked in primary caveolinopathy, suggesting a subset of these patients may also possess mutations within proteins required for membrane targeting of dysferlin.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Caveolin 1 / genetics
  • Caveolin 1 / metabolism
  • Cohort Studies
  • Cytoplasm / metabolism
  • Cytoplasm / pathology
  • DNA Mutational Analysis
  • Dysferlin
  • Gene Frequency
  • Genetic Predisposition to Disease / genetics*
  • Genetic Testing
  • Humans
  • Membrane Proteins / genetics*
  • Membrane Proteins / metabolism
  • Muscle Fibers, Skeletal / metabolism
  • Muscle Fibers, Skeletal / pathology
  • Muscle Proteins / genetics*
  • Muscle Proteins / metabolism
  • Muscle, Skeletal / metabolism
  • Muscle, Skeletal / pathology*
  • Muscle, Skeletal / physiopathology*
  • Muscular Dystrophies, Limb-Girdle / classification
  • Muscular Dystrophies, Limb-Girdle / diagnosis*
  • Muscular Dystrophies, Limb-Girdle / genetics*
  • Mutation / genetics
  • Protein Transport / genetics
  • Regeneration / genetics
  • Retrospective Studies
  • Sarcolemma / metabolism
  • Sarcolemma / pathology

Substances

  • Caveolin 1
  • DYSF protein, human
  • Dysferlin
  • Membrane Proteins
  • Muscle Proteins