Screening and detection of gene mutations in Japanese patients with Fabry disease by non-radioactive single-stranded conformation polymorphism analysis

Brain Dev. 1997 Mar;19(2):111-6. doi: 10.1016/s0387-7604(96)00486-x.

Abstract

We have applied non-radioactive polymerase chain reaction (PCR)-single-stranded conformation polymorphism (SSCP) to the detection of gene mutations causing Fabry disease. Nineteen of 22 known mutations were detected as electrophoretic mobility shifts on PCR-SSCP analysis. Then, DNA from newly diagnosed Japanese patients with the classical form of Fabry disease was subjected to PCR-SSCP analysis, and 4 novel mutations (1 small deletion, 1 nonsense mutation and 2 missense mutations) and 1 neutral polymorphism were identified. Furthermore, identification of an asymptomatic heterozygote and a hemizygote with moderate clinical manifestations was successfully achieved by application of this method to a family with the variant form of Fabry disease. PCR-SSCP is useful for the gene diagnosis of etiologically heterogeneous Fabry disease.

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • Fabry Disease / genetics*
  • Female
  • Genetic Testing / methods*
  • Heterozygote
  • Humans
  • Japan
  • Male
  • Middle Aged
  • Mutation
  • Pedigree
  • Polymerase Chain Reaction
  • Polymorphism, Single-Stranded Conformational*
  • alpha-Galactosidase / genetics

Substances

  • alpha-Galactosidase