The first nonsense mutation in alsin results in a homogeneous phenotype of infantile-onset ascending spastic paralysis with bulbar involvement in two siblings

Clin Genet. 2003 Sep;64(3):210-5. doi: 10.1034/j.1399-0004.2003.00138.x.

Abstract

Eight mutations in the ALS2 gene have been described as causing autosomal-recessive juvenile-onset forms of the motor neuron diseases amyotrophic lateral sclerosis, primary lateral sclerosis and hereditary spastic paraplegia. All mutations are small deletions that are predicted to result in a frameshift and premature truncation of the alsin protein. Here we describe a ninth ALS2 mutation, in two siblings affected by infantile-onset ascending spastic paraplegia with bulbar involvement. This mutation is predicted to result in the substitution of an amino acid by a stop codon, and thus is the first nonsense mutation detected in this gene. It is probable that full-length alsin is required for the proper development and/or functioning of upper motor neurons.

Publication types

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

MeSH terms

  • Age of Onset
  • Codon, Nonsense*
  • Consanguinity
  • Exons / genetics
  • Female
  • Humans
  • Infant
  • Intellectual Disability / genetics
  • Jews / genetics
  • Pedigree
  • Phenotype
  • Protein Isoforms / genetics
  • Protein Structure, Tertiary
  • Pseudobulbar Palsy / genetics*
  • Pseudobulbar Palsy / pathology
  • Spastic Paraplegia, Hereditary / epidemiology
  • Spastic Paraplegia, Hereditary / genetics*
  • Spastic Paraplegia, Hereditary / pathology

Substances

  • Codon, Nonsense
  • Protein Isoforms