Scientists test One-Shot genetic cure for devastating blood disease
⭐️ CURE ⭐️
Completed
This early-stage study tested a potential one-time treatment for adults with severe sickle cell disease. Doctors collected a patient's own blood stem cells, used a modified virus to insert a healthy gene into them, and then gave them back to the patient. The goal was to see if th…
Phase: PHASE1, PHASE2 • Sponsor: Donald B. Kohn, M.D. • Aim: ⭐️ CURE ⭐️
Last updated Mar 13, 2026 15:05 UTC