Gene therapy offers hope for 'Bubble Boy' disease, but leukemia risk remains
NCT ID NCT01129544
First seen Apr 22, 2026 · Last updated May 22, 2026 · Updated 4 times
Summary
This study tested a gene therapy for children with SCID-X1, a severe immune disorder, who lack a matched donor. Doctors inserted a normal gene into the child's own bone marrow stem cells to fix the immune system without chemotherapy. Eight children were treated, and while many saw immune improvement, some needed additional treatment and there was a risk of leukemia, a serious side effect seen in earlier trials.
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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.
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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