Unexpected and variable phenotypes in a family with JAK3 deficiency

Genes Immun. 2001 Dec;2(8):422-32. doi: 10.1038/sj.gene.6363802.

Abstract

Mutations of the Janus kinase 3 (JAK3) have been previously described to cause an autosomal recessive variant of severe combined immunodeficiency (SCID) usually characterized by the near absence of T and NK cells, but preserved numbers of B lymphocytes (T-B+SCID). We now report a family whose JAK3 mutations are associated with the persistence of circulating T cells, resulting in previously undescribed clinical presentations, ranging from a nearly unaffected 18-year-old subject to an 8-year-old sibling with a severe lymphoproliferative disorder. Both siblings were found to be compound heterozygotes for the same deleterious JAK3 mutations: an A96G initiation start site mutation, resulting in a dysfunctional, truncated protein product and a G2775(+3)C mutation in the splice donor site sequence of intron 18, resulting in a splicing defect and a predicted premature stop. These mutations were compatible with minimal amounts of functional JAK3 expression, leading to defective cytokine-dependent signaling. Activated T cells in these patients failed to express Fas ligand (FasL) in response to IL-2, which may explain the accumulation of T cells with an activated phenotype and a skewed T cell receptor (TcR) Vbeta family distribution. We speculate that residual JAK3 activity accounted for the maturation of thymocytes, but was insufficient to sustain IL-2-mediated homeostasis of peripheral T cells via Fas/FasL interactions. These data demonstrate that the clinical spectrum of JAK3 deficiency is quite broad and includes immunodeficient patients with accumulation of activated T cells, and indicate an essential role for JAK3 in the homeostasis of peripheral T cells in humans.

MeSH terms

  • Adolescent
  • Amino Acid Sequence
  • B-Lymphocytes / metabolism
  • Cell Line, Transformed
  • Child
  • DNA, Complementary
  • Fas Ligand Protein
  • Female
  • Gene Expression
  • Humans
  • Immunophenotyping
  • Interleukin-2 / metabolism
  • Janus Kinase 3
  • Male
  • Membrane Glycoproteins / metabolism
  • Molecular Sequence Data
  • Mutation
  • Pedigree
  • Protein-Tyrosine Kinases / deficiency*
  • Protein-Tyrosine Kinases / genetics
  • Receptors, Antigen, T-Cell, alpha-beta / metabolism
  • Receptors, Interleukin-2 / metabolism
  • Sequence Analysis, DNA
  • Severe Combined Immunodeficiency / genetics
  • Severe Combined Immunodeficiency / immunology
  • Severe Combined Immunodeficiency / pathology
  • Signal Transduction
  • T-Lymphocytes
  • Up-Regulation

Substances

  • DNA, Complementary
  • FASLG protein, human
  • Fas Ligand Protein
  • Interleukin-2
  • Membrane Glycoproteins
  • Receptors, Antigen, T-Cell, alpha-beta
  • Receptors, Interleukin-2
  • Protein-Tyrosine Kinases
  • JAK3 protein, human
  • Janus Kinase 3