The intron 7 donor splice site transition: a second Tay-Sachs disease mutation in French Canada

Hum Genet. 1992 Dec;90(4):402-6. doi: 10.1007/BF00220467.

Abstract

Mutations at the hexosaminidase A (HEXA) gene which cause Tay-Sachs disease (TSD) have elevated frequency in the Ashkenazi Jewish and French-Canadian populations. We report a novel TSD allele in the French-Canadian population associated with the infantile form of the disease. The mutation, a G-->A transition at the +1 position of intron 7, abolishes the donor splice site. Cultured human fibroblasts from a compound heterozygote for this transition (and for a deletion mutation) produce no detectable HEXA mRNA. The intron 7 + 1 mutation occurs in the base adjacent to the site of the adult-onset TSD mutation (G805A). In both mutations a restriction site for the endonuclease EcoRII is abolished. Unambiguous diagnosis, therefore, requires allele-specific oligonucleotide hybridization to distinguish between these two mutant alleles. The intron 7 + 1 mutation has been detected in three unrelated families. Obligate heterozygotes for the intron 7 + 1 mutation were born in the Saguenay-Lac-St-Jean region of Quebec. The most recent ancestors common to obligate carriers of this mutation were from the Charlevoix region of the province of Quebec. This mutation thus has a different geographic centre of diffusion and is probably less common than the exon 1 deletion TSD mutation in French Canadians. Neither mutation has been detected in France, the ancestral homeland of French Canada.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Base Sequence
  • DNA, Single-Stranded
  • Female
  • Gene Expression
  • Gene Frequency
  • Heterozygote
  • Hexosaminidase A
  • Humans
  • Introns*
  • Male
  • Molecular Sequence Data
  • Mutation
  • Pedigree
  • Polymerase Chain Reaction
  • Quebec
  • RNA Splicing*
  • RNA, Messenger / metabolism
  • Tay-Sachs Disease / genetics*
  • beta-N-Acetylhexosaminidases / genetics

Substances

  • DNA, Single-Stranded
  • RNA, Messenger
  • Hexosaminidase A
  • beta-N-Acetylhexosaminidases