Gene therapy offers hope for bubble boy disease, but leukemia risk remains
NCT ID NCT01129544
First seen Apr 22, 2026 · Last updated May 15, 2026 · Updated 3 times
Summary
This study tested a gene therapy for children with SCID-X1, a severe immune disorder, who lacked a matched donor. The approach used the child's own bone marrow stem cells, adding a normal gene to fix the immune system without chemotherapy or transplant complications. Eight children were treated; some achieved immune improvement, but a serious side effect of leukemia occurred in previous trials, highlighting ongoing risks.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Children's Hospital Boston
Boston, Massachusetts, 02116, United States
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Cincinnati Children's Medical Center
Cincinnati, Ohio, 45229, United States
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Mattel Children's Hospital - UCLA
Los Angeles, California, 90095, United States
Conditions
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