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


ÖZEN 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 (SCI-Expanded) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 22 Issue: 2
  • Publication Date: 2021
  • Doi Number: 10.1038/s41590-020-00830-z
  • Journal Name: NATURE IMMUNOLOGY
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, BIOSIS, Biotechnology Research Abstracts, CAB Abstracts, Chemical Abstracts Core, EMBASE, MEDLINE, Veterinary Science Database
  • Page Numbers: pp.128-151
  • Çukurova University Affiliated: Yes

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.