A double-variant transthyretin allele (Ser 6, Ile 33) in the Israeli patient "SKO" with familial amyloidotic polyneuropathy

Hum Mutat. 1994;3(3):254-60. doi: 10.1002/humu.1380030313.

Abstract

Transthyretin (TTR) isolated from amyloid fibrils from an Israeli patient ("SKO") with familial amyloidotic polyneuropathy has been studied by two groups of investigators. Originally, a position 49 Thr-->Gly substitution was reported; subsequently, a position 33 Phe-->Ile substitution was found instead. We have studied DNA from this patient by single strand conformation polymorphism analysis, restriction analysis, and DNA sequencing. On one allele, exon 2 contained both a T-->A transversion at the first position of codon 33, encoding the previously described Phe-->Ile substitution, and a G-->A transition at the first position of codon 6, encoding a Gly-->Ser substitution. The originally reported position 49 mutation was not encoded in the genomic DNA. This is the first report of a TTR double-variant allele in a patient with TTR amyloidosis.

Publication types

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

MeSH terms

  • Alleles
  • Amino Acid Sequence
  • Amyloid Neuropathies / genetics*
  • Base Sequence
  • DNA / genetics
  • DNA / isolation & purification
  • DNA Primers
  • Exons
  • Humans
  • Introns
  • Isoleucine*
  • Israel
  • Molecular Sequence Data
  • Point Mutation*
  • Polymerase Chain Reaction / methods
  • Polymorphism, Genetic
  • Prealbumin / genetics*
  • Restriction Mapping
  • Serine*

Substances

  • DNA Primers
  • Prealbumin
  • Isoleucine
  • Serine
  • DNA