Uncoordinated HLA-D gene expression in a RFXANK-defective patient with MHC class II deficiency

J Immunol. 2001 May 1;166(9):5681-7. doi: 10.4049/jimmunol.166.9.5681.

Abstract

We describe the analysis of a patient, JER, presenting classical immunological features of MHC class II deficiency. Unexpectedly, some HLA transcripts (HLA-DRA, HLA-DQA, and HLA-DMA) were found to be expressed in the JER cell line at nearly wild-type levels, while HLA-DPA and the HLA-D beta-chain transcripts were not detected. Gene reporter experiments confirmed the differential transcriptional activities driven by the HLA-D promoters in the JER cells. A defect in RFXANK was first suggested by genetic complementation analyses, then assessed with the demonstration of a homozygous mutation affecting a splice donor site downstream exon 4 of RFXANK. Because the severe deletion of the resulting protein cannot account for the expression of certain HLA-D genes, minor alternative transcripts of the RFXANK gene were analyzed. We thereby showed the existence of a transcript lacking exon 4, encoding a 28-aa-deleted protein that retains a transcriptional activity. Altogether, we characterize a new type of mutation in the RFXANK gene in a MHC class II-defective patient leading to an uncoordinated expression of the HLA-D genes, and propose that this phenotype is ensured by severely limited amounts of an active, although truncated RFXANK protein.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alternative Splicing / genetics
  • Alternative Splicing / immunology
  • B-Lymphocytes / immunology
  • B-Lymphocytes / metabolism
  • Cell Line, Transformed
  • DNA-Binding Proteins
  • Female
  • Gene Expression Regulation / immunology*
  • Genes, MHC Class II*
  • Genetic Complementation Test
  • HLA-D Antigens / biosynthesis
  • HLA-D Antigens / genetics*
  • HLA-DQ Antigens / biosynthesis
  • HLA-DQ Antigens / genetics
  • HLA-DQ alpha-Chains
  • HLA-DR Antigens / biosynthesis
  • HLA-DR Antigens / genetics
  • HLA-DR alpha-Chains
  • Humans
  • Infant
  • Male
  • RNA, Messenger / biosynthesis
  • Sequence Deletion
  • Severe Combined Immunodeficiency / genetics*
  • Severe Combined Immunodeficiency / immunology*
  • Transcription Factors / biosynthesis
  • Transcription Factors / deficiency*
  • Transcription Factors / genetics*

Substances

  • DNA-Binding Proteins
  • HLA-D Antigens
  • HLA-DQ Antigens
  • HLA-DQ alpha-Chains
  • HLA-DQA1 antigen
  • HLA-DR Antigens
  • HLA-DR alpha-Chains
  • RFXANK protein, human
  • RNA, Messenger
  • Transcription Factors