Doctors test One-Time gene fix for rare blood disorder in kids
NCT ID NCT04248439
Summary
This study is testing a one-time gene therapy for children with Fanconi Anemia type A, a rare genetic disorder that causes bone marrow failure. Doctors take the patient's own blood stem cells, correct the faulty gene in a lab, and then return the corrected cells to the patient. The goal is to prevent or delay the need for bone marrow transplants by helping the body produce healthy blood cells.
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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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Stanford University
Stanford, California, 94304, United States
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University of Minnesota
Minneapolis, Minnesota, 55454, United States
Conditions
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