Evidence for defects in V(D)J rearrangements in patients with severe combined immunodeficiency

J Immunol. 1994 Jun 1;152(11):5504-13.

Abstract

We investigated the patterns of DNA rearrangements at loci for Ig JH genes in patients with severe combined immunodeficiency (SCID). Four SCID patients without B cells (B- SCID) and four SCID patients with B cells (B+ SCID) were examined. Bone marrow cells of these patients were transformed with EBV. The majority of the transformed cells from three B- SCID patients had the germline configuration at their JH gene loci. The rearranged fragments from one patient were analyzed extensively. The rearranged regions in all of the fragments had a common structure wherein two fragments derived from the JH-S mu region were connected inversely. The possible presence of rearranged forms of VHDJH and DHQ52JH sequences in bone marrow cells of two B- SCID patients were examined directly by the polymerase chain reaction (PCR) method. In one patient, we found neither a VHDJH sequence nor a DHQ52JH sequence within the range of sensitivity of the PCR method. In another patient, we found a VHDJH sequence at an extremely low level and DHQ52JH sequences at a relatively low level. Either RAG-1 or RAG-2 gene was not expressed in the B- SCID-derived cell lines. B+ SCID patients did not show any abnormalities in terms of VHDJH rearrangements. These results indicate that B- SCID may be caused by defects in factors involved in V(D)J rearrangements.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Base Sequence
  • Cell Line
  • Cell Line, Transformed
  • DNA-Binding Proteins*
  • Female
  • Gene Rearrangement*
  • Genes, Immunoglobulin*
  • Homeodomain Proteins*
  • Humans
  • Immunoglobulin Joining Region / genetics*
  • Immunoglobulin Variable Region / genetics*
  • Infant
  • Male
  • Molecular Sequence Data
  • Nuclear Proteins
  • Polymerase Chain Reaction
  • Proteins / genetics
  • Severe Combined Immunodeficiency / genetics*
  • Severe Combined Immunodeficiency / immunology

Substances

  • DNA-Binding Proteins
  • Homeodomain Proteins
  • Immunoglobulin Joining Region
  • Immunoglobulin Variable Region
  • Nuclear Proteins
  • Proteins
  • RAG2 protein, human
  • V(D)J recombination activating protein 2
  • RAG-1 protein

Associated data

  • GENBANK/S71044
  • GENBANK/S71045
  • GENBANK/S71046
  • GENBANK/S71047