X-LINKED SEVERE COMBINED IMMUNODEFICIENCY
Clinical trials for X-LINKED SEVERE COMBINED IMMUNODEFICIENCY explained in plain language.
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Gene therapy offers hope for 'Bubble Boy' disease, but leukemia risk remains
Disease control CompletedThis 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…
Matched conditions: X-LINKED SEVERE COMBINED IMMUNODEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: David Williams • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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Gene therapy offers hope for boys with severe immune disorder
Disease control CompletedThis trial tested a gene therapy for X-linked severe combined immunodeficiency (SCID-X1), a life-threatening condition where boys are born without a working immune system. Five boys who had no matching bone marrow donor received a single infusion of their own blood stem cells tha…
Matched conditions: X-LINKED SEVERE COMBINED IMMUNODEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC