Novel compound heterozygous AIRE mutations in a Japanese patient with APECED

J Pediatr Endocrinol Metab. 2004 Jun;17(6):917-21. doi: 10.1515/jpem.2004.17.6.917.

Abstract

Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED) is a rare autosomal recessive disorder defined by the presence of two of three conditions, namely, Addison's disease, hypoparathyroidism, and mucocutaneous candidiasis. APECED is caused by alteration in a single gene, named the autoimmune regulator (AIRE) gene. We report AIRE gene mutations in a Japanese female with APECED. The patient is a 22-year-old Japanese female who was diagnosed with Addison's disease, hypoparathyroidism, and mucocutaneous candidiasis at age 8 years. Sequence analysis of the AIRE gene revealed novel compound heterozygous mutations. One was 1471 delCinsTT in exon 11 (GenBank accession no. AB006682), which leads to a frameshift and premature truncation of a 502 amino acid protein. The other was a G-->A transition at IVS11+1. Her mother was heterozygous for 1471 delCinsTT and was normal homozygous for IVS11+1. We found novel compound heterozygous mutations in the AIRE gene of a Japanese female with APECED.

Publication types

  • Case Reports

MeSH terms

  • AIRE Protein
  • Addison Disease / genetics
  • Adult
  • Asian People / genetics*
  • Base Sequence
  • Candidiasis, Chronic Mucocutaneous / genetics*
  • Cytosine
  • Ectodermal Dysplasia / genetics*
  • Exons
  • Female
  • Frameshift Mutation
  • Gene Deletion
  • Heterozygote
  • Humans
  • Hypoparathyroidism / genetics
  • Mutagenesis, Insertional
  • Mutation*
  • Polyendocrinopathies, Autoimmune / genetics*
  • Syndrome
  • Thymidine
  • Transcription Factors / genetics*

Substances

  • Transcription Factors
  • Cytosine
  • Thymidine