Gene therapy trial offers hope for boys with rare immune disease
NCT ID NCT01855685
First seen Apr 26, 2026 · Last updated May 19, 2026 · Updated 5 times
Summary
This study tested a gene therapy for boys with X-linked chronic granulomatous disease (X-CGD), a rare disorder where white blood cells cannot fight infections. The treatment involved taking the patient's own blood stem cells, adding a corrected gene, and returning them to the body. The goal was to restore immune function and reduce 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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
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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