Homozygosity for the transthyretin-Met30 gene in three Japanese siblings with type I familial amyloidotic polyneuropathy

Neurology. 1992 Oct;42(10):2045-7. doi: 10.1212/wnl.42.10.2045.

Abstract

We report the cases of three siblings homozygous for a mutated transthyretin (TTR) gene that causes type I familial amyloidotic polyneuropathy (FAP), in whom we made the diagnosis by identifying both the mutated TTR gene and a variant TTR in their sera. Their serum levels for the variant TTR are twice those of patients heterozygous for the gene, but two have late-onset FAP and the third is an elderly asymptomatic carrier. TTR abnormality is a necessary condition for the development of FAP, but there may be other factors that retard or prevent its clinical development.

Publication types

  • Case Reports

MeSH terms

  • Aged
  • Amyloid / genetics*
  • Amyloid / metabolism
  • Amyloidosis / blood
  • Amyloidosis / genetics*
  • Chromatography, High Pressure Liquid
  • DNA / analysis
  • Female
  • Genes*
  • Homozygote*
  • Humans
  • Male
  • Molecular Probes / genetics
  • Mutation
  • Nervous System Diseases / blood
  • Nervous System Diseases / genetics*
  • Pedigree
  • Polymerase Chain Reaction
  • Prealbumin / genetics*
  • Prealbumin / metabolism

Substances

  • Amyloid
  • Molecular Probes
  • Prealbumin
  • amyloid prealbumin
  • DNA