Doctors test One-Time gene fix for rare blood disorder in kids
NCT ID NCT04248439
Summary
This trial is testing a one-time gene therapy for children with a rare, serious genetic disorder called Fanconi anemia type A. Doctors take a patient's own blood stem cells, fix the faulty gene in a lab, and then return the corrected cells to the patient. The goal is to prevent the bone marrow failure that is common with this disease.
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Contacts and locations
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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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