Homozygosity for the transthyretin-met30-gene in two Swedish sibs with familial amyloidotic polyneuropathy

Clin Genet. 1988 Nov;34(5):333-8. doi: 10.1111/j.1399-0004.1988.tb02887.x.

Abstract

Familial amyloidotic polyneuropathy (FAP) is an autosomal dominant inherited disorder. Recent biochemical studies have revealed that amyloid protein in FAP of Japanese, Swedish and Portuguese origin mainly consists of a variant transthyretin (TTR) (formerly called prealbumin) with one amino acid substitution of methionine for valine at position 30. In a 56-year-old man with typical polyneuropathy, gastrointestinal problems and vitreous amyloid, we diagnosed homozygosity for the TTR-met30-gene using RFLP analysis. In a family study, a sister presented the same homozygous RFLP pattern; however, in a careful clinical investigation we were not able to demonstrate any of the typical symptoms of FAP, nor could we demonstrate amyloid deposits in a biopsy skin specimen. This is the first report of homozygosity for the TTR-met30-gene, and it shows that the mutation of the protein involved in amyloid formation may be necessary but is clearly not sufficient for the clinical symptoms.

Publication types

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

MeSH terms

  • Adult
  • Amyloidosis / genetics*
  • Female
  • Hereditary Sensory and Autonomic Neuropathies / genetics*
  • Hereditary Sensory and Motor Neuropathy / genetics*
  • Homozygote
  • Humans
  • Male
  • Methionine / genetics*
  • Middle Aged
  • Mutation
  • Pedigree
  • Prealbumin / analysis
  • Prealbumin / biosynthesis
  • Prealbumin / genetics*
  • Sweden

Substances

  • Prealbumin
  • Methionine