RNase H2 catalytic core Aicardi-Goutières syndrome-related mutant invokes cGAS-STING innate immune-sensing pathway in mice

J Exp Med. 2016 Mar 7;213(3):329-36. doi: 10.1084/jem.20151464. Epub 2016 Feb 15.

Abstract

The neuroinflammatory autoimmune disease Aicardi-Goutières syndrome (AGS) develops from mutations in genes encoding several nucleotide-processing proteins, including RNase H2. Defective RNase H2 may induce accumulation of self-nucleic acid species that trigger chronic type I interferon and inflammatory responses, leading to AGS pathology. We created a knock-in mouse model with an RNase H2 AGS mutation in a highly conserved residue of the catalytic subunit, Rnaseh2a(G37S/G37S) (G37S), to understand disease pathology. G37S homozygotes are perinatal lethal, in contrast to the early embryonic lethality previously reported for Rnaseh2b- or Rnaseh2c-null mice. Importantly, we found that the G37S mutation led to increased expression of interferon-stimulated genes dependent on the cGAS-STING signaling pathway. Ablation of STING in the G37S mice results in partial rescue of the perinatal lethality, with viable mice exhibiting white spotting on their ventral surface. We believe that the G37S knock-in mouse provides an excellent animal model for studying RNASEH2-associated autoimmune diseases.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, N.I.H., Intramural

MeSH terms

  • Animals
  • Autoimmune Diseases of the Nervous System / genetics
  • Autoimmune Diseases of the Nervous System / immunology*
  • Catalytic Domain
  • Cells, Cultured
  • Crosses, Genetic
  • Embryo, Mammalian / metabolism
  • Female
  • Fibroblasts / metabolism
  • Gene Expression Regulation
  • HEK293 Cells
  • Homozygote
  • Humans
  • Immunity, Innate*
  • Interferons / metabolism
  • Long Interspersed Nucleotide Elements / genetics
  • Male
  • Membrane Proteins / metabolism*
  • Mice
  • Mutation / genetics*
  • Nervous System Malformations / genetics
  • Nervous System Malformations / immunology*
  • Nucleotidyltransferases / metabolism*
  • Phenotype
  • Ribonuclease H / chemistry*
  • Ribonuclease H / metabolism*
  • Signal Transduction

Substances

  • Membrane Proteins
  • Sting1 protein, mouse
  • Interferons
  • Nucleotidyltransferases
  • cGAS protein, mouse
  • Ribonuclease H

Supplementary concepts

  • Aicardi-Goutieres syndrome