Mutations in phenotypically mild D-2-hydroxyglutaric aciduria

Ann Neurol. 2005 Oct;58(4):626-30. doi: 10.1002/ana.20559.

Abstract

D-2-hydroxyglutaric aciduria is a neurometabolic disorder with mild and severe phenotypes. Recently, we reported pathogenic mutations in the D-2-hydroxyglutarate dehydrogenase gene as the cause of the severe phenotype of D-2-hydroxyglutaric aciduria in two patients. Here, we report two novel pathogenic mutations in this gene in one patient with a mild presentation and two asymptomatic siblings with D-2-hydroxyglutaric aciduria from two unrelated consanguineous Palestinian families: a splice error (IVS4-2A-->G) and a missense mutation (c.1315A-->G;p.Asn439Asp). Overexpression of this mutant protein showed marked reduction of the enzyme activity.

Publication types

  • Case Reports

MeSH terms

  • Alcohol Oxidoreductases / genetics*
  • Alcohol Oxidoreductases / urine*
  • Amino Acid Metabolism, Inborn Errors / genetics*
  • DNA, Recombinant
  • Family Health
  • Female
  • Humans
  • Male
  • Mutation*
  • Phenotype*

Substances

  • DNA, Recombinant
  • Alcohol Oxidoreductases
  • 2-hydroxyglutarate dehydrogenase