Scientists test One-Time gene fix for 'Bubble Boy' disease
NCT ID NCT06253507
Summary
This study is testing a new gene therapy for people with a rare, inherited immune disorder called p47 chronic granulomatous disease (CGD). Doctors will collect a patient's own blood stem cells, add a corrected copy of the missing gene in a lab, and then return the modified cells to the patient. The goal is to see if this one-time treatment can help the immune system fight infections better and for a long time.
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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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