Scientists try gene editing to fix rare 'Bubble Boy' immune disease
NCT ID NCT06559176
First seen Apr 14, 2026 · Last updated Apr 14, 2026
Summary
This early-stage study is testing a new gene-editing treatment for people with a rare, inherited immune disorder called chronic granulomatous disease (CGD). Doctors will collect a patient's own blood stem cells, use a precise editing tool to try to fix the genetic error causing the disease, and then return the corrected cells to the patient. The main goals are to see if this process is safe and if it can restore the immune system's ability to fight infections.
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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.