Aicardi-Goutières syndrome (AGS)

Eur J Paediatr Neurol. 2008 Sep;12(5):355-8. doi: 10.1016/j.ejpn.2007.11.010. Epub 2008 Mar 14.

Abstract

In 1984, Jean Aicardi and Françoise Goutières described 8 children showing both severe brain atrophy and chronic cerebrospinal fluid lymphocytosis, with basal ganglia calcification in at least one member of each affected family. The course was rapid to death or a vegetative outcome. Aicardi and Goutières correctly predicted that the disorder would be genetic, but emphasised that "some features, especially the pleocytosis, may erroneously suggest an inflammatory condition". The increased interferon-alpha in affected children (Pierre Lebon, Paris) mimicked congenital viral infection, but the associated chilblains (pernio) pointed to lupus erythematosus and an autoimmune mechanism. Genetic research led by Yanick Crow has clarified these puzzling relationships in Aicardi-Goutières syndrome, a syndrome that now includes conditions previously known as microcephaly-intracranial calcification syndrome, pseudo-TORCH and Cree encephalitis. At the time of writing, Crow's team has discovered that over 80% of families with Aicardi-Goutières syndrome have mutations in one of four nuclease genes, the exonuclease TREX1 and the genes for all three subunits of the ribonuclease H2 enzyme complex. Aicardi-Goutières syndrome is both genetically and phenotypically heterogeneous, with a range of severity from life-threatening perinatal illness to mild late infancy onset. All infants of whatever genotype have increased interferon-alpha in the first year of life and this appears to be the final common pathway that links Aicardi-Goutières syndrome, congenital virus infection and systemic lupus erythematosus.

Publication types

  • Review

MeSH terms

  • Autoimmune Diseases of the Nervous System / enzymology*
  • Autoimmune Diseases of the Nervous System / genetics*
  • Autoimmune Diseases of the Nervous System / physiopathology
  • Basal Ganglia Diseases / enzymology*
  • Basal Ganglia Diseases / genetics*
  • Basal Ganglia Diseases / physiopathology
  • Calcinosis / enzymology
  • Calcinosis / genetics
  • Calcinosis / physiopathology
  • Diagnosis, Differential
  • Exodeoxyribonucleases / genetics*
  • Genetic Predisposition to Disease / genetics*
  • Humans
  • Infant
  • Infant, Newborn
  • Interferon-alpha / genetics
  • Lymphocytosis / cerebrospinal fluid
  • Phosphoproteins / genetics*
  • Ribonuclease H / genetics
  • Syndrome

Substances

  • Interferon-alpha
  • Phosphoproteins
  • Exodeoxyribonucleases
  • three prime repair exonuclease 1
  • ribonuclease HII
  • Ribonuclease H