Identical mutations in RAG1 or RAG2 genes leading to defective V(D)J recombinase activity can cause either T-B-severe combined immune deficiency or Omenn syndrome

Blood. 2001 May 1;97(9):2772-6. doi: 10.1182/blood.v97.9.2772.

Abstract

Omenn syndrome (OS) is an inherited disorder characterized by an absence of circulating B cells and an infiltration of the skin and the intestine by activated oligoclonal T lymphocytes, indicating that a profound defect in the lymphoid developmental program could be accountable for this condition. Inherited mutations in either the recombination activating genes RAG1 or RAG2, resulting in partial V(D)J recombinase activity, were shown to be responsible for OS. This study reports on the characterization of new RAG1/2 gene mutations in a series of 9 patients with OS. Given the occurrence of the same mutations in patients with T-B-severe combined immune deficiency or OS on 3 separate occasions, the proposal is made that an additional factor may be required in certain circumstances for the development of the Omenn phenotype. The nature of this factor is discussed.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • DNA Nucleotidyltransferases / genetics
  • DNA Nucleotidyltransferases / metabolism
  • DNA-Binding Proteins*
  • Female
  • Gene Expression Regulation, Enzymologic / immunology
  • Genes, RAG-1*
  • Humans
  • Infant
  • Male
  • Mutation*
  • Nuclear Proteins
  • Severe Combined Immunodeficiency / enzymology
  • Severe Combined Immunodeficiency / etiology
  • Severe Combined Immunodeficiency / genetics*
  • Syndrome
  • VDJ Recombinases

Substances

  • DNA-Binding Proteins
  • Nuclear Proteins
  • RAG2 protein, human
  • V(D)J recombination activating protein 2
  • DNA Nucleotidyltransferases
  • VDJ Recombinases