V(D)J recombination defects in lymphocytes due to RAG mutations: severe immunodeficiency with a spectrum of clinical presentations

Blood. 2001 Jan 1;97(1):81-8. doi: 10.1182/blood.v97.1.81.

Abstract

Severe combined immunodeficiency (SCID) comprises a heterogeneous group of primary immunodeficiencies, a proportion of which are due to mutations in either of the 2 recombination activating genes (RAG)-1 and -2, which mediate the process of V(D)J recombination leading to the assembly of antigen receptor genes. It is reported here that the clinical and immunologic phenotypes of patients bearing mutations in RAGs are more diverse than previously thought and that this variability is related, in part, to the specific type of RAG mutation. By analyzing 44 such patients from 41 families, the following conclusions were reached: (1) null mutations on both alleles lead to the T-B-SCID phenotype; (2) patients manifesting classic Omenn syndrome (OS) have missense mutations on at least one allele and maintain partial V(D)J recombination activity, which accounts for the generation of residual, oligoclonal T-lymphocytes; (3) in a third group of patients, findings were only partially compatible with OS, and these patients, who also carried at least one missense mutation, may be considered to have atypical SCID/OS; (4) patients with engraftment of maternal T cells as a complication of a transplacental transfusion represented a fourth group, and these patients, who often presented with a clinical phenotype mimicking OS, may be observed regardless of the type of RAG gene mutation. Analysis of the RAG genes by direct sequencing is an effective way to provide accurate diagnosis of RAG-deficient as opposed to RAG-independent V(D)J recombination defects, a distinction that cannot be made based on clinical and immunologic phenotype alone.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alleles
  • Cohort Studies
  • DNA Mutational Analysis
  • DNA-Binding Proteins / genetics
  • Databases, Factual
  • Family Health
  • Female
  • Genes, RAG-1 / genetics*
  • Genotype
  • Humans
  • Immunoglobulin Joining Region / genetics*
  • Immunoglobulin Variable Region / genetics*
  • Immunophenotyping
  • Infant
  • Infant, Newborn
  • Lymphocytes / immunology*
  • Lymphopenia / etiology
  • Male
  • Maternal-Fetal Exchange / immunology
  • Mutation
  • Mutation, Missense
  • Nuclear Proteins
  • Pregnancy
  • Recombination, Genetic
  • Severe Combined Immunodeficiency / complications
  • Severe Combined Immunodeficiency / genetics
  • T-Lymphocytes / transplantation

Substances

  • DNA-Binding Proteins
  • Immunoglobulin Joining Region
  • Immunoglobulin Variable Region
  • Nuclear Proteins
  • RAG2 protein, human
  • V(D)J recombination activating protein 2