One-Time treatment aims to cure devastating immune disorder
NCT ID NCT07113743
Summary
This study is testing a one-time gene therapy for people with X-linked Chronic Granulomatous Disease (X-CGD), a rare genetic disorder that cripples the immune system. Doctors will collect a patient's own blood stem cells, use a virus to insert a healthy copy of the faulty gene, and then return the corrected cells after chemotherapy. The goal is to create a lasting, functional immune system to fight off severe infections.
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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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