The immunopathological landscape of human pre-TCRα deficiency: From rare to common variants

Science. 2024 Mar;383(6686):eadh4059. doi: 10.1126/science.adh4059. Epub 2024 Mar 1.

Abstract

We describe humans with rare biallelic loss-of-function PTCRA variants impairing pre-α T cell receptor (pre-TCRα) expression. Low circulating naive αβ T cell counts at birth persisted over time, with normal memory αβ and high γδ T cell counts. Their TCRα repertoire was biased, which suggests that noncanonical thymic differentiation pathways can rescue αβ T cell development. Only a minority of these individuals were sick, with infection, lymphoproliferation, and/or autoimmunity. We also report that 1 in 4000 individuals from the Middle East and South Asia are homozygous for a common hypomorphic PTCRA variant. They had normal circulating naive αβ T cell counts but high γδ T cell counts. Although residual pre-TCRα expression drove the differentiation of more αβ T cells, autoimmune conditions were more frequent in these patients compared with the general population.

MeSH terms

  • Aged
  • Aged, 80 and over
  • Alleles
  • Autoimmunity* / genetics
  • Cell Differentiation
  • Female
  • Homozygote
  • Humans
  • Infections / immunology
  • Intraepithelial Lymphocytes* / immunology
  • Loss of Function Mutation
  • Lymphocyte Count
  • Lymphoproliferative Disorders / immunology
  • Male
  • Membrane Glycoproteins* / genetics
  • Middle Aged
  • Pedigree
  • Receptors, Antigen, T-Cell, alpha-beta* / genetics

Substances

  • Receptors, Antigen, T-Cell, alpha-beta
  • pre-T cell receptor alpha
  • Membrane Glycoproteins