Fumarase deficiency is an autosomal recessive encephalopathy affecting both the mitochondrial and the cytosolic enzymes

Neurology. 1990 Mar;40(3 Pt 1):495-9. doi: 10.1212/wnl.40.3_part_1.495.

Abstract

A 7-month-old boy died in a demented state after a clinical history characterized by generalized seizures, psychomotor deterioration, and fumaric aciduria. We found a marked deficiency of both mitochondrial and cytosolic fumarases in skeletal muscle, brain, cerebellum, heart, kidney, liver, and cultured fibroblasts. Fumarase activities were 30 to 50% compared with controls in both mitochondria and cytosol from cultured fibroblasts of the parents. Antifumarase cross-reacting material was present in negligible amounts in the patient's tissues. Our data indicate that this disease is an autosomal recessive encephalopathy, due to a single mutation affecting the gene encoding both forms of the enzyme.

Publication types

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

MeSH terms

  • Acids / blood
  • Acids / urine
  • Blotting, Western
  • Brain Diseases, Metabolic / enzymology
  • Brain Diseases, Metabolic / genetics*
  • Carnitine / blood
  • Carnitine / urine
  • Chromatography, Gas
  • Chromosome Aberrations / metabolism
  • Chromosome Disorders
  • Cytosol / enzymology*
  • Cytosol / metabolism
  • Fumarate Hydratase / deficiency*
  • Genes, Recessive
  • Humans
  • Infant
  • Isoenzymes / metabolism
  • Male
  • Mitochondria / enzymology*
  • Mitochondria / metabolism

Substances

  • Acids
  • Isoenzymes
  • Fumarate Hydratase
  • Carnitine