Broadly effective metabolic and immune recovery with C5 inhibition in CHAPLE disease


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Ozen A. O. , Kasap N., Vujkovic-Cvijin I., Apps R., Cheung F., Karakoc-Aydiner E. , ...More

NATURE IMMUNOLOGY, vol.22, no.2, pp.128-151, 2021 (Journal Indexed in SCI) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 22 Issue: 2
  • Publication Date: 2021
  • Doi Number: 10.1038/s41590-020-00830-z
  • Title of Journal : NATURE IMMUNOLOGY
  • Page Numbers: pp.128-151

Abstract

CHAPLE disease is a lethal syndrome caused by genetic loss of the complement regulatory protein CD55. Lenardo, Ozen and their colleagues report that blockade of C5 complement activation in a small cohort of pediatric patients with CHAPLE disease reduced gastrointestinal pathology and restored their immunity and growth.