Missense mutation serine106----proline causes 17 alpha-hydroxylase deficiency

J Biol Chem. 1991 Aug 25;266(24):15992-8.

Abstract

Steroid 17 alpha-hydroxylase deficiency is caused by defects in cytochrome P450c17, the single enzyme that has 17-alpha hydroxylase and 17,20-lyase activities. We describe a rapid and efficient polymerase chain reaction tactic for identifying these genetic lesions and identify Ser106----Pro as the cause of 17 alpha-hydroxylase deficiency in two unrelated homozygous patients from Guam. We used site-directed mutagenesis of the normal P450c17 cDNA to construct the Pro106 mutant, and expressed both the normal and mutant sequences in monkey COS-1 cells and in yeast. Expression of the normal sequence permitted the cells to convert pregnenolone to 17-OH pregnenolone, progesterone to 17-OH progesterone, and 17-OH pregnenolone to dehydroepiandrosterone, showing the normal sequence conferred both 17 alpha-hydroxylase and 17,20-lyase activities. Expression of the mutant sequence generated P450c17 mRNA, but conferred none of these activities, proving that the Ser106----Pro mutation abolished the 17 alpha-hydroxylase and 17,20-lyase activities. An HhaI restriction site created by the mutation should permit screening of large populations.

Publication types

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

MeSH terms

  • Adolescent
  • Adrenal Hyperplasia, Congenital*
  • Alleles
  • Base Sequence
  • Blotting, Northern
  • Blotting, Southern
  • Chromatography, Thin Layer
  • DNA / genetics
  • Electrophoresis, Polyacrylamide Gel
  • Female
  • Homozygote
  • Humans
  • Molecular Sequence Data
  • Mutagenesis, Site-Directed
  • Mutation*
  • Polymerase Chain Reaction
  • Pregnenolone / metabolism
  • Progesterone / metabolism
  • Proline / chemistry*
  • RNA / genetics
  • Serine / chemistry*
  • Steroid 17-alpha-Hydroxylase / genetics

Substances

  • Serine
  • Progesterone
  • RNA
  • Pregnenolone
  • DNA
  • Proline
  • Steroid 17-alpha-Hydroxylase

Associated data

  • GENBANK/M62906
  • GENBANK/M62907
  • GENBANK/S51169
  • GENBANK/S51174
  • GENBANK/S51178
  • GENBANK/S51183
  • GENBANK/S51188
  • GENBANK/S51192
  • GENBANK/S51674
  • GENBANK/S51705