N-terminal truncated human RAG1 proteins can direct T-cell receptor but not immunoglobulin gene rearrangements

Blood. 2000 Jul 1;96(1):203-9.

Abstract

The proteins encoded by RAG1 and RAG2 can initiate gene recombination by site-specific cleavage of DNA in immunoglobulin and T-cell receptor (TCR) loci. We identified a new homozygous RAG1 gene mutation (631delT) that leads to a premature stop codon in the 5' part of the RAG1 gene. The patient carrying this 631delT RAG1 gene mutation died at the age of 5 weeks from an Omenn syndrome-like T(+)/B(- )severe combined immunodeficiency disease. The high number of blood T-lymphocytes (55 x 10(6)/mL) showed an almost polyclonal TCR gene rearrangement repertoire not of maternal origin. In contrast, B-lymphocytes and immunoglobulin gene rearrangements were hardly detectable. We showed that the 631delT RAG1 gene can give rise to an N-terminal truncated RAG1 protein, using an internal AUG codon as the translation start site. Consistent with the V(D)J recombination in T cells, this N-terminal truncated RAG1 protein was active in a plasmid V(D)J recombination assay. Apparently, the N-terminal truncated RAG1 protein can recombine TCR genes but not immunoglobulin genes. We conclude that the N-terminus of the RAG1 protein is specifically involved in immunoglobulin gene rearrangements.

Publication types

  • Case Reports

MeSH terms

  • B-Lymphocytes / immunology
  • Codon, Terminator
  • Consanguinity
  • Fatal Outcome
  • Female
  • Gene Rearrangement*
  • Gene Rearrangement, T-Lymphocyte*
  • Genes, Immunoglobulin*
  • Genes, RAG-1*
  • Homeodomain Proteins / genetics*
  • Homeodomain Proteins / immunology*
  • Homozygote
  • Humans
  • Immunologic Deficiency Syndromes / genetics*
  • Immunologic Deficiency Syndromes / immunology
  • Immunophenotyping
  • Infant, Newborn
  • Male
  • Sequence Deletion
  • T-Lymphocytes / immunology*

Substances

  • Codon, Terminator
  • Homeodomain Proteins
  • RAG-1 protein