L: -2-Hydroxyglutaric aciduria, a disorder of metabolite repair

J Inherit Metab Dis. 2009 Apr;32(2):135-42. doi: 10.1007/s10545-008-1042-3. Epub 2008 Nov 21.

Abstract

The neurometabolic disorder L: -2-hydroxyglutaric aciduria is caused by mutations in a gene present on chromosome 14q22.1 and encoding L: -2-hydroxyglutarate dehydrogenase. This FAD-linked mitochondrial enzyme catalyses the irreversible conversion of L: -2-hydroxyglutarate to alpha-ketoglutarate. The formation of L: -2-hydroxyglutarate results from a side-activity of mitochondrial L: -malate dehydrogenase, the enzyme that interconverts oxaloacetate and L: -malate, but which also catalyses, very slowly, the NADH-dependent conversion of alpha-ketoglutarate to L: -2-hydroxyglutarate. L: -2-Hydroxyglutarate has no known physiological function in eukaryotes and most prokaryotes. Its accumulation is toxic to the mammalian brain, causing a leukoencephalopathy and increasing the susceptibility to develop tumours. L: -2-Hydroxyglutaric aciduria appears to be the first disease of 'metabolite repair'.

Publication types

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

MeSH terms

  • Alcohol Oxidoreductases / genetics*
  • Amino Acid Metabolism, Inborn Errors / enzymology*
  • Amino Acid Metabolism, Inborn Errors / genetics*
  • Amino Acid Metabolism, Inborn Errors / physiopathology
  • Flavin-Adenine Dinucleotide / metabolism
  • Glutarates / urine*
  • Humans
  • NAD / metabolism
  • Nervous System Diseases / enzymology*
  • Nervous System Diseases / genetics*
  • Nervous System Diseases / physiopathology

Substances

  • Glutarates
  • NAD
  • Flavin-Adenine Dinucleotide
  • alpha-hydroxyglutarate
  • Alcohol Oxidoreductases
  • 2-hydroxyglutarate dehydrogenase