A novel form of complete IL-12/IL-23 receptor beta1 deficiency with cell surface-expressed nonfunctional receptors

Blood. 2004 Oct 1;104(7):2095-101. doi: 10.1182/blood-2004-02-0584. Epub 2004 Jun 3.

Abstract

Complete interleukin-12/interleukin-23 receptor beta1 (IL-12Rbeta1) deficiency is the most frequent known genetic etiology of the syndrome of Mendelian susceptibility to mycobacterial disease. The patients described to date lack IL-12Rbeta1 at the surface of their natural killer (NK) and T cells due to IL12RB1 mutations, which either interrupt the open reading frame or disrupt protein folding. We describe a patient with a large in-frame deletion of 12165 nucleotides (nt) in IL12RB1, encompassing exons 8 to 13 and resulting in the surface expression of nonfunctional IL-12Rbeta1. These 6 exons encode the proximal NH2-terminal half of the extracellular domain downstream from the cytokine-binding domain. Five of 6 monoclonal anti-IL-12Rbeta1 antibodies tested recognized the internally truncated chain on the cell surface. However, IL-12 and IL-23 did not bind normally to the patient's IL-12Rbeta1-containing respective heterodimeric receptors. As a result, signal transducer and activator of transcription-4 (STAT4) was not phosphorylated and interferon-gamma (IFN-gamma) production was not induced in the patient's cells upon stimulation with even high doses of IL-12 or IL-23. The functional defect was completely rescued by retrovirus-mediated IL-12Rbeta1 gene transfer. Thus, the detection of IL-12Rbeta1 on the cell surface does not exclude the possibility of complete IL-12Rbeta1 deficiency in patients with mycobacteriosis or salmonellosis. Paradoxically, the largest IL12RB1 mutation detected is associated with the cell surface expression of nonfunctional IL-12Rbeta1, defining a novel genetic form of IL-12Rbeta1 deficiency.

Publication types

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

MeSH terms

  • Antibodies, Monoclonal / chemistry
  • Cell Membrane / metabolism*
  • Cells, Cultured
  • Child
  • Cytokines / metabolism
  • DNA, Complementary / metabolism
  • DNA-Binding Proteins / metabolism
  • Enzyme-Linked Immunosorbent Assay
  • Exons
  • Flow Cytometry
  • Gene Deletion
  • Gene Transfer Techniques
  • Humans
  • Interferon-gamma / metabolism
  • Interleukin-12 / metabolism
  • Interleukin-23
  • Interleukin-23 Subunit p19
  • Interleukins / metabolism*
  • Killer Cells, Natural / metabolism
  • Male
  • Models, Genetic
  • Mutation
  • Open Reading Frames
  • Phenotype
  • Phosphorylation
  • Polymerase Chain Reaction
  • Protein Folding
  • Protein Structure, Tertiary
  • RNA / metabolism
  • Receptors, Interleukin / deficiency
  • Receptors, Interleukin / metabolism*
  • Receptors, Interleukin-12
  • Retroviridae / genetics
  • STAT4 Transcription Factor
  • Time Factors
  • Trans-Activators / metabolism

Substances

  • Antibodies, Monoclonal
  • Cytokines
  • DNA, Complementary
  • DNA-Binding Proteins
  • IL12RB1 protein, human
  • IL23A protein, human
  • Interleukin-23
  • Interleukin-23 Subunit p19
  • Interleukins
  • Receptors, Interleukin
  • Receptors, Interleukin-12
  • STAT4 Transcription Factor
  • STAT4 protein, human
  • Trans-Activators
  • Interleukin-12
  • RNA
  • Interferon-gamma