Phenotypic variability of aprataxin gene mutations

Neurology. 2003 Mar 11;60(5):868-70. doi: 10.1212/01.wnl.0000048562.88536.a4.

Abstract

The clinical and genetic features of three non-Portuguese and non-Japanese patients with aprataxin gene mutations are reported. Patient 1 came from Italy and presented with typical ataxia with ocular motor apraxia (OMA). She was homozygous for the W279X nonsense mutation, which is associated with the Portuguese founding haplotype. Patients 2 and 3 were French siblings and did not present with either OMA or hypoalbuminemia. They were compound heterozygous for the nonsense W279X mutation and a missense K197Q mutation.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Apraxias / genetics*
  • Ataxia / genetics*
  • Atrophy / genetics
  • Cerebellum / pathology
  • Codon, Nonsense*
  • DNA-Binding Proteins / genetics*
  • Female
  • Humans
  • Male
  • Mutation, Missense*
  • Nervous System Diseases / genetics*
  • Nuclear Proteins / genetics*
  • Ocular Motility Disorders / genetics*
  • Peripheral Nervous System Diseases / diagnosis
  • Peripheral Nervous System Diseases / genetics
  • Phenotype
  • Syndrome

Substances

  • APTX protein, human
  • Codon, Nonsense
  • DNA-Binding Proteins
  • Nuclear Proteins