Gene therapy offers hope for rare immune disorder cure
NCT ID NCT03837483
Summary
This trial tests a gene therapy that aims to cure Wiskott-Aldrich Syndrome, a rare genetic disorder that weakens the immune system. Doctors collect patients' own blood stem cells, add a corrected gene in the lab, then return them to the patient. The study measures whether this treatment reduces severe infections and bleeding episodes compared to before treatment.
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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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