Gene therapy offers hope for 'Bubble Boy' disease, but leukemia risk remains
Disease control
Completed
This trial tested a gene therapy for children with SCID-X1, a severe immune disorder, who do not have a matched bone marrow donor. The treatment involves harvesting the child's own bone marrow cells, fixing the broken gene in the lab, and infusing the corrected cells back after m…
Phase: PHASE1, PHASE2 • Sponsor: David Williams • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC