Residual activity of human porphobilinogen deaminase with R167Q or R167W mutations: an explanation for survival of homozygous and compound heterozygous acute intermittent porphyrics

Cell Mol Biol (Noisy-le-grand). 2002 Dec;48(8):861-6.

Abstract

To find an explanation for survival of homozygous or compound heterozygous variants of acute intermittent porphyria, we studied the three mutant forms of porphobilinogen deaminase (PBG-d) described in the four reported patients with homozygous acute intermittent porphyria. Wild-type human PBG-d and the PBG-d R167W, R167Q and R173Q mutants were expressed in Escherichia coli and the recombinant mutant human enzyme were examined for enzyme activity. Specific antibodies against human PBG-d detected the three human PBG-d mutants. All three had less than 2% of wild-type enzyme activity when examined under customary assay conditions (pH 8.0), but the R167W and R167Q mutants were found to have about 25% of normal activity when assayed at pH 7.0. This residual activity at a more physiological pH provides an explanation for survival when these mutations are inherited in a homozygous or compound heterozygous fashion.

MeSH terms

  • Dose-Response Relationship, Drug
  • Electrophoresis, Polyacrylamide Gel
  • Enzyme-Linked Immunosorbent Assay
  • Escherichia coli / genetics
  • Escherichia coli / metabolism
  • Heterozygote
  • Homozygote
  • Humans
  • Hydrogen-Ion Concentration
  • Hydroxymethylbilane Synthase / genetics*
  • Hydroxymethylbilane Synthase / metabolism*
  • Isoelectric Focusing
  • Liver / metabolism
  • Mutation*
  • Porphyria, Acute Intermittent / genetics*
  • Porphyria, Acute Intermittent / mortality*
  • Recombinant Proteins / metabolism

Substances

  • Recombinant Proteins
  • Hydroxymethylbilane Synthase