Gene therapy trial offers hope for boys with rare immune disease
NCT ID NCT02757911
First seen Apr 21, 2026 · Last updated May 15, 2026 · Updated 4 times
Summary
This study tested a gene therapy for boys with X-linked chronic granulomatous disease (X-CGD), a rare disorder that leaves them unable to fight off certain infections. The treatment involved taking the patients' own blood stem cells, adding a working copy of the faulty gene, and returning them to the body. The goal was to restore immune function and reduce severe infections and inflammation. Only 3 participants were enrolled before the study was terminated.
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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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Locations
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Hôpital Necker Enfants Malades
Paris, 75015, France
Conditions
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