Scientists test One-Shot genetic fix for devastating blood disease
NCT ID NCT03964792
Summary
This early-stage study tested a one-time gene therapy for people with severe sickle cell disease. Doctors collected a patient's own blood stem cells, used a modified virus to insert a healthy gene into them in the lab, and then gave the corrected cells back to the patient. The goal was to see if this procedure was safe and could produce healthy red blood cells, potentially curing the disease.
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Contacts and locations
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Locations
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Department of Biotherapy, Necker-Enfants Malades Hospital
Paris, 75015, France
Conditions
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