Early step toward a gene therapy cure for sickle cell?
NCT ID NCT02212535
Summary
This small, early-stage study tested the safety and effectiveness of a single injection of a drug called plerixafor. The goal was to see if it could safely move stem cells from the bone marrow into the bloodstream of adults with severe sickle cell disease, so those cells could be collected. These collected stem cells could potentially be used in future gene therapy treatments aimed at controlling the disease.
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Contacts and locations
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Locations
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Hôpital Necker - Enfants Malades
Paris, 75015, France
Conditions
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