One-Time treatment aims to free patients from lifelong blood transfusions
NCT ID NCT04390971
Summary
This early study tested a one-time gene therapy called ET-01 in three young patients with severe transfusion-dependent thalassemia. The goal was to see if modifying a patient's own blood stem cells could help their body produce healthy hemoglobin, reducing or eliminating the need for regular blood transfusions. Researchers monitored safety and whether the modified cells successfully engrafted and began working.
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Contacts and locations
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Locations
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Affiliated Hospital of Zunyi Medical University
Zunyi, Guizhou, 563000, China
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Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020, China
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The Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, 550004, China
Conditions
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