L-2-hydroxyglutaric aciduria: characterisation of the molecular defect in a spontaneous canine model

J Med Genet. 2007 May;44(5):334-40. doi: 10.1136/jmg.2006.042507.

Abstract

l-2-hydroxyglutaric aciduria (l-2-HGA) is a neurometabolic disorder that produces a variety of clinical neurological deficits, including psychomotor retardation, seizures and ataxia. The biochemical hallmark of l-2-HGA is the accumulation of l-2-hydroxyglutaric acid (l-2-HG) in cerebrospinal fluid, plasma and urine. Mutations within the gene L2HGDH (Entrez Gene ID 79944) on chromosome 14q22 encoding L-2-hydroxyglutaric acid dehydrogenase have recently been shown to cause l-2-HGA in humans. Using a candidate gene approach in an outbred pet dog population segregating l-2-HGA, the causal molecular defect was identified in the canine homologue of L2HGDH and characterised. DNA sequencing and pedigree analysis indicate a common founder effect in the canine model. The canine model shares many of the clinical and MRI features of the disease in humans and represents a valuable resource as a spontaneous model of l-2-HGA.

Publication types

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

MeSH terms

  • Amino Acid Metabolism, Inborn Errors / enzymology
  • Amino Acid Metabolism, Inborn Errors / genetics*
  • Amino Acid Metabolism, Inborn Errors / veterinary*
  • Amino Acid Sequence
  • Animals
  • Base Pairing / genetics
  • Base Sequence
  • Brain / diagnostic imaging
  • DNA Mutational Analysis
  • Disease Models, Animal
  • Dog Diseases / enzymology
  • Dog Diseases / genetics*
  • Dogs
  • Exons / genetics
  • Glutarates / metabolism*
  • Glutarates / urine
  • Glutaryl-CoA Dehydrogenase / chemistry
  • Glutaryl-CoA Dehydrogenase / genetics
  • Heterozygote
  • Homozygote
  • Magnetic Resonance Imaging
  • Microsatellite Repeats / genetics
  • Molecular Sequence Data
  • Mutation / genetics
  • Pedigree
  • Radiography

Substances

  • Glutarates
  • alpha-hydroxyglutarate
  • Glutaryl-CoA Dehydrogenase