Lack of iNKT cells in patients with combined immune deficiency due to hypomorphic RAG mutations

Blood. 2008 Jan 1;111(1):271-4. doi: 10.1182/blood-2007-06-096487. Epub 2007 Sep 21.

Abstract

Hypomorphic mutations of the RAG genes in humans are associated with a spectrum of clinical and immunologic presentations that range from T(-) B(-) severe combined immune deficiency (SCID) to Omenn syndrome. In most cases, residual V(D)J recombination activity allows for development of few T-cell clones, which expand in the periphery and infiltrate target organs, resulting in tissue damage. Invariant natural killer T (iNKT) cells play an important immunoregulatory role and have been associated with protection against autoimmunity. We now report on 5 unrelated cases of combined immune deficiency due to hypomorphic RAG mutations, and demonstrate the absence of iNKT cells in all 5 patients. These findings suggest that lack of this important immunoregulatory cell population may contribute to the pathophysiology of Omenn syndrome.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Line, Tumor
  • Homeodomain Proteins / genetics*
  • Humans
  • Immunophenotyping
  • Infant
  • Infant, Newborn
  • Killer Cells, Natural / immunology
  • Killer Cells, Natural / pathology*
  • Point Mutation
  • Severe Combined Immunodeficiency / genetics*
  • Severe Combined Immunodeficiency / immunology
  • Severe Combined Immunodeficiency / pathology*
  • T-Lymphocytes / immunology

Substances

  • Homeodomain Proteins
  • RAG-1 protein