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

ÖZEN, AHMET; Kasap, Nurhan; Vujkovic-Cvijin, Ivan; Apps, Richard; Cheung, Foo; Karakoc-Aydiner, Elif; Akkelle, Bilge; SARI, SİNAN; TUTAR, ENGİN; Ozcay, Figen; Uygun, Dilara; İŞLEK, ALİ; Akgun, Gamze; Selcuk, Merve; Sezer, Oya; Zhang, Yu; Kutluk, Gunsel; Topal, Erdem; Sayar, Ersin; Celikel, Cigdem; Houwen, Roderick; Bingol, Aysen; Ogulur, Ismail; Eltan, Sevgi; Snow, Andrew; Lake, Camille; Fantoni, Giovanna; Alba, Camille; Sellers, Brian; Chauvin, Samuel; Dalgard, Clifton; Harari, Olivier; Ni, Yan; Wang, Ming-Dauh; Devalaraja-Narashimha, Kishor; Subramanian, Poorani; Ergelen, Rabia; Artan, Reha; Guner, Sukru; Dalgic, Buket; Tsang, John; Belkaid, Yasmine; Ertem, Deniz; Baris, Safa; Lenardo, Michael

doi:10.1038/s41590-020-00830-z

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

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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)

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.