Partial ornithine transcarbamylase deficiency in females: diagnosis by an immunohistochemical method

Eur J Pediatr. 1987 Jul;146(4):370-2. doi: 10.1007/BF00444940.

Abstract

Females heterozygous for the X-linked urea cycle disorder, ornithine transcarbamylase (OTC) deficiency have a significant risk of developing hyperammonaemia. Diagnosis of this genetic defect in a proband is the essential starting point for family studies. By an immunohistochemical analysis of the liver specimens fixed in 10% formalin, we confirmed heterozygous status for OTC deficiency in two female patients, a 15-year-old girl and a 2-year-old girl, who died of hyperammonaemia. Since most affected males lack cross reactive materials (CRM), an immunochemical analysis should be useful for the diagnosis of most heterozygous females.

Publication types

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

MeSH terms

  • Adolescent
  • Amino Acid Metabolism, Inborn Errors / genetics*
  • Amino Acid Metabolism, Inborn Errors / pathology
  • Ammonia / blood*
  • Carbamoyl-Phosphate Synthase (Ammonia) / metabolism
  • Child, Preschool
  • Female
  • Genetic Carrier Screening
  • Genetic Linkage
  • Humans
  • Immunoenzyme Techniques*
  • Liver / pathology
  • Ornithine Carbamoyltransferase Deficiency Disease*
  • Sex Chromosome Aberrations / genetics
  • X Chromosome

Substances

  • Ammonia
  • Carbamoyl-Phosphate Synthase (Ammonia)