Mutations in senataxin responsible for Quebec cluster of ataxia with neuropathy

Ann Neurol. 2005 Mar;57(3):408-14. doi: 10.1002/ana.20408.

Abstract

Senataxin recently was identified as the mutated gene in ataxia-oculomotor apraxia 2, which is characterized by ataxia, oculomotor apraxia, and increased alpha-fetoprotein levels. In this study, we evaluated 24 ataxic patients from 10 French-Canadian families. All cases have a homogeneous phenotype consisting of a progressive ataxia appearing between 2 and 20 (mean age, 14.8) years of age with associated dysarthria, saccadic ocular pursuit, distal amyotrophy, sensory and motor neuropathy, and increased alpha-fetoprotein levels but absence of oculomotor apraxia. Linkage disequilibrium was observed with markers in the ataxia-oculomotor apraxia 2 locus on chromosome 9q34. We have identified four mutations in senataxin in the French-Canadian population including two novel missense mutations: the 5927T-->G mutation changes the leucine encoded by codon 1976 to an arginine in the helicase domain (L1976R), and the 193G-->A mutation changes a glutamic acid encoded by codon 65 into a lysine in the N-terminal domain of the protein (E65K). The common L1976R mutation is shared by 17 of 20 (85%) carrier chromosomes. The study of this large French-Canadian cohort better defines the phenotype of this ataxia and presents two novel mutations in senataxin including the more common founder mutation in the French-Canadian population.

Publication types

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

MeSH terms

  • Adult
  • Aged
  • Apraxias / complications
  • Apraxias / genetics
  • Arginine / genetics
  • Ataxia / complications
  • Ataxia / genetics*
  • Chromosomes, Human, Pair 9
  • Cluster Analysis*
  • DNA Helicases
  • DNA Mutational Analysis / methods
  • Family Health
  • Female
  • Glutamic Acid / genetics
  • Humans
  • Leucine / genetics
  • Linkage Disequilibrium
  • Lysine / genetics
  • Male
  • Middle Aged
  • Multifunctional Enzymes
  • Mutation / genetics*
  • Oculomotor Nerve Diseases / complications
  • Oculomotor Nerve Diseases / genetics*
  • Quebec
  • RNA Helicases / genetics*
  • alpha-Fetoproteins / metabolism

Substances

  • Multifunctional Enzymes
  • alpha-Fetoproteins
  • Glutamic Acid
  • Arginine
  • SETX protein, human
  • DNA Helicases
  • RNA Helicases
  • Leucine
  • Lysine