Gene therapy offers hope for rare immune disorder
NCT ID NCT03837483
First seen Nov 01, 2025 · Last updated Apr 24, 2026 · Updated 21 times
Summary
This study tests a gene therapy for people with Wiskott-Aldrich syndrome, a rare genetic disease that causes frequent infections and bleeding. The treatment uses the patient's own blood stem cells, which are modified in a lab to fix the faulty gene and then returned to the body. The goal is to reduce severe infections and bleeding episodes compared to before treatment.
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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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Children's Healthcare of Atlanta, Inc
Atlanta, Georgia, 30329, United States
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Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Milan, 20132, Italy
Conditions
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