Scientists test One-Time gene fix for devastating blood disorder
NCT ID NCT03282656
Summary
This is an early safety study testing a one-time gene therapy for people with severe sickle cell disease. Doctors collect a patient's own blood stem cells, use a modified virus to insert a corrective gene, and then return the cells after chemotherapy. The goal is to see if this approach is safe and can increase healthy fetal hemoglobin to control the disease.
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Contacts and locations
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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UCLA - Mattel Children's Hospital
Los Angeles, California, 90095, United States
Conditions
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