Skewed T-cell receptor repertoire, decreased thymic output, and predominance of terminally differentiated T cells in ataxia telangiectasia

Blood. 2002 Dec 1;100(12):4082-9. doi: 10.1182/blood-2002-03-0976. Epub 2002 Aug 8.

Abstract

Ataxia telangiectasia (A-T), a genetic disorder caused by the homozygous mutation of the ATM gene, frequently associates with variable degrees of cellular and humoral immunodeficiency. However, the immune defects occurring in patients with A-T are still poorly characterized. Here we show that the T-cell receptor (TCR) variable beta (BV)-chain repertoire of 9 A-T patients was restricted by diffuse expansions of some variable genes prevalently occurring within the CD4 subset and clustering to certain TCRBV genes (eg, 5.1, 11, 14, and 23). In addition, the study of the third complementarity-determining region (CDR3) showed, in all patients, significantly altered profiles in most BV genes examined suggesting diffuse oligoclonal expansions. The sequencing of TCR CDR3 regions revealed completely normal V(D)J coding joints and confirmed a reduced diversity of the antigen-receptor repertoire. The B-cell repertoire was similarly restricted and skewed by diffuse oligoclonal expansions with normal V(D)J joints. Thymic output, evaluated by measuring TCR rearrangement excision circles, was extremely low. The majority of peripheral T cells had the phenotype and the function of effector memory cells, indicating that in vivo they are able to respond normally by terminal differentiation to antigenic stimulation. These results indicate that ATM mutation limits the generation of a wide repertoire of normally functioning T and B cells.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Amino Acid Sequence
  • Ataxia Telangiectasia / pathology*
  • Cell Differentiation
  • Child
  • Complementarity Determining Regions / genetics
  • Female
  • Gene Rearrangement, beta-Chain T-Cell Antigen Receptor
  • Humans
  • Immunoglobulin Heavy Chains
  • Immunoglobulin Variable Region
  • Immunophenotyping
  • Male
  • Receptors, Antigen, T-Cell / genetics*
  • T-Lymphocytes / immunology
  • T-Lymphocytes / pathology*
  • Thymus Gland / immunology*
  • Thymus Gland / pathology

Substances

  • Complementarity Determining Regions
  • Immunoglobulin Heavy Chains
  • Immunoglobulin Variable Region
  • Receptors, Antigen, T-Cell