Scientists test One-Shot gene fix for blood disorder
NCT ID NCT06024876
Summary
This early-stage study tested a new one-time treatment for beta-thalassemia, a serious inherited blood disorder. Researchers collected a small number of patients' own blood stem cells, used gene editing to 'switch on' a protective fetal hemoglobin gene, and then returned the cells. The goal was to see if this could safely reduce or eliminate the need for lifelong blood transfusions.
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Contacts and locations
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Locations
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The First Affiliated Hospital of Guangxi Medical University
Nanning, China
Conditions
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