Rewriting the code: a One-Time gene fix for a rare immune disease?
⭐️ CURE ⭐️
Not yet recruiting
This trial tests a one-time gene therapy for WHIM syndrome, a rare inherited immune disorder that causes severe infections. Doctors collect a person's own blood stem cells, use base editing to correct the faulty CXCR4 gene, and return the edited cells to the body. The goal is to …
Phase: PHASE1, PHASE2 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: ⭐️ CURE ⭐️
Last updated Aug 23, 2026 00:00 UTC