Clinical and molecular findings in patients with giant axonal neuropathy (GAN)

Neurology. 2004 Jan 13;62(1):13-6. doi: 10.1212/01.wnl.0000101676.41505.a7.

Abstract

Giant axonal neuropathy (GAN) is a rare autosomal recessive neurodegenerative disorder of early onset, clinically characterized by a progressive involvement of both peripheral and CNS. The diagnosis is based on the presence of characteristic giant axons, filled with neurofilaments, on nerve biopsy. Recently, the defective protein, gigaxonin, has been identified and different pathogenic mutations in the gigaxonin gene have been reported as the underlying genetic defect. Gigaxonin, a member of the BTB/kelch superfamily proteins, seems to play a crucial role in the cross talk between the intermediate filaments and the membrane network. The authors report clinical and molecular findings in five Italian patients with GAN. This study shows the allelic heterogeneity of GAN and expands the spectrum of mutations in the GAN gene. The frequent occurrence of private mutations stresses the importance of a complete gene analysis.

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • Ataxia / complications
  • Ataxia / diagnosis
  • Ataxia / genetics
  • Axons / pathology*
  • Child
  • Child, Preschool
  • Cognition Disorders / complications
  • Cognition Disorders / diagnosis
  • Cognition Disorders / genetics
  • Cytoskeletal Proteins / genetics*
  • DNA Mutational Analysis
  • Disease Progression
  • Electromyography
  • Exons
  • Female
  • Humans
  • Introns
  • Italy
  • Male
  • Mutation
  • Neurodegenerative Diseases / complications
  • Neurodegenerative Diseases / diagnosis*
  • Neurodegenerative Diseases / genetics*
  • Neurofilament Proteins
  • Sural Nerve / pathology

Substances

  • Cytoskeletal Proteins
  • GAN protein, human
  • Neurofilament Proteins

Associated data

  • OMIM/256850

Grants and funding