Gene therapy breakthrough offers hope for boys with rare immune disease
NCT ID NCT01855685
First seen Apr 26, 2026 · Last updated May 13, 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 corrected gene, and returning them to the body. The goal was to restore the immune system's ability to kill bacteria and fungi, reducing severe infections and inflammation.
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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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Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom
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University College London Hospital (UCLH)
London, NW1 2PG, United Kingdom
Conditions
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