Expansion of GAA triplet repeats in the human genome: unique origin of the FRDA mutation at the center of an Alu

Genomics. 2004 Mar;83(3):373-83. doi: 10.1016/j.ygeno.2003.09.001.

Abstract

Friedreich ataxia is caused by expansion of a GAA triplet repeat (GAA-TR) in the FRDA gene. Normal alleles contain <30 triplets, and disease-causing expansions (66-1700 triplets) arise via hyperexpansion of premutations (30-65 triplets). To gain insight into GAA-TR instability we analyzed all triplet repeats in the human genome. We identified 988 (GAA)(8+) repeats, 291 with >or=20 triplets, including 29 potential premutations (30-62 triplets). Most other triplet repeats were restricted to <20 triplets. We estimated the expected frequency of (GAA)(6+) repeats to be negligible, further indicating that GAA-TRs have undergone significant expansion. Eighty-nine percent of (GAA)(8+) sequences map within G/A islands, and 58% map within the poly(A) tails of Alu elements. Only two other (GAA)(8+) sequences shared the central Alu location seen at the FRDA locus. One showed allelic variation, including expansions analogous to short Friedreich ataxia mutations. Our data demonstrate that GAA-TRs have expanded throughout primate evolution with the generation of potential premutation alleles at multiple loci.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Algorithms
  • Alu Elements / genetics*
  • Base Sequence
  • Evolution, Molecular
  • Frataxin
  • Friedreich Ataxia / genetics
  • Gene Frequency
  • Genome, Human*
  • Humans
  • Iron-Binding Proteins / genetics*
  • Models, Genetic
  • Mutation*
  • Trinucleotide Repeat Expansion*

Substances

  • Iron-Binding Proteins