Gene editing breakthrough offers hope for rare immune disease
NCT ID NCT06559176
First seen Apr 14, 2026 · Last updated May 17, 2026 · Updated 5 times
Summary
This study tests a new gene therapy for people with chronic granulomatous disease (CGD), a rare immune disorder that causes severe infections. The treatment uses the patient's own blood stem cells, which are modified in a lab with a precise gene-editing tool called prime editing, then returned to the body. The goal is to restore the immune system's ability to fight infections. About 12 participants will be followed for 15 years to check safety and effectiveness.
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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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CHU - Sainte Justine Hospital
Montreal, Quebec, H3T 1C5, Canada
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NIH Clinical Center
Bethesda, Maryland, 20892, United States
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The Children's Hospital at Tristar Medical Group/Sarah Cannon Center for Blood Cancers
Nashville, Tennessee, 37203, United States
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University College of London Hospital
London, England, NW1 2PG, United Kingdom
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University of California Los Angeles Medical Center
Los Angeles, California, 90027, United States
Conditions
Explore the condition pages connected to this study.