X-chromosome inactivation analysis in a female carrier of FOXP3 mutation

Clin Exp Immunol. 2002 Oct;130(1):127-30. doi: 10.1046/j.1365-2249.2002.01940.x.

Abstract

Immune dysregulation, polyendocrinopathy and enteropathy with X-linked inheritance (IPEX) is a serious disease arising from mutations in FOXP3. This gene codifies for a transcription factor whose dysfunction results in hyperactivation of T cells. It is not clear, however, why an intermediate phenotype is not seen in heterozygous females, who are completely healthy. In order to address this question, we investigated X-chromosome inactivation in peripheral blood lymphocytes from a heterozygous female with a child affected by IPEX. No preferential inactivation was shown in freshly sorted CD4+, CD8+, CD19+ cells or in IL-2 cultured CD4 and CD8 T cells, indicating that peripheral blood lymphocytes in these women are randomly selected. Moreover, only one single FOXP3 transcript was expressed by CD4 T cell clones analysed by RT-PCR, confirming that this gene is subject to X- inactivation. We hypothesize that hyper-activation of T cell in carriers of FOXP3 mutations is regulated by the presence of normal regulatory T cells.

Publication types

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

MeSH terms

  • Adult
  • Amino Acid Substitution
  • Child
  • DNA Mutational Analysis
  • DNA-Binding Proteins / genetics*
  • DNA-Binding Proteins / physiology
  • Diabetes Mellitus, Type 1 / genetics
  • Diarrhea / genetics
  • Dosage Compensation, Genetic*
  • Endocrine System Diseases / genetics*
  • Female
  • Forkhead Transcription Factors
  • Genotype
  • Heterozygote
  • Humans
  • Lymphocyte Activation
  • Lymphocyte Subsets / immunology
  • Lymphocyte Subsets / pathology*
  • Lymphoproliferative Disorders / genetics*
  • Male
  • Mutation, Missense
  • Point Mutation
  • Syndrome

Substances

  • DNA-Binding Proteins
  • FOXP3 protein, human
  • Forkhead Transcription Factors