Complete FXN deletion in a patient with Friedreich's ataxia

Genet Test Mol Biomarkers. 2012 Sep;16(9):1015-8. doi: 10.1089/gtmb.2012.0012. Epub 2012 Jun 12.

Abstract

Most patients (98%) with Friedreich's ataxia (FRDA) are homozygous for the GAA repeat expansion in FXN. Only a few compound heterozygous patients with an expanded repeat on one allele and a point mutation or an intragenic FXN deletion on the other allele are described. In a minority of the patients only a heterozygous pattern of the repeat expansion can be detected. Using array analysis after GAA repeat expansion testing, we identified a FRDA patient who is compound heterozygous for an expanded GAA repeat and a complete FXN deletion. Since not only repeat expansions and point mutations, but also large rearrangements can be the underlying cause of FRDA, a quantitative test should also be performed in case a patient shows only one allele with an expanded GAA repeat in FXN.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Female
  • Frataxin
  • Friedreich Ataxia / genetics*
  • Gene Deletion*
  • Heterozygote*
  • Humans
  • Iron-Binding Proteins / genetics*
  • Male
  • Pedigree
  • Trinucleotide Repeat Expansion / genetics*

Substances

  • Iron-Binding Proteins