Gene editing offers new hope for 'Bubble Boy' disease
NCT ID NCT06851767
First seen Nov 18, 2025 · Last updated Apr 29, 2026 · Updated 17 times
Summary
This study tests a new gene therapy for X-linked severe combined immunodeficiency (XSCID), a rare immune disorder. Researchers collect a person's own stem cells, fix the faulty gene using base editing, and return the cells to the body. The goal is to rebuild the immune system. The study includes 18 people aged 3 and older and follows them for 15 years.
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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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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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