One-Time gene therapy aims to cure inherited blood disorder
NCT ID NCT06328764
Summary
This early-stage study is testing a one-time treatment called CS-101 for people with beta-thalassemia, a serious inherited blood disorder. Doctors will collect a patient's own blood stem cells, edit a specific gene in the lab to boost healthy fetal hemoglobin production, and then return the cells to the patient. The goal is to see if this treatment is safe and can free patients from needing regular blood transfusions, potentially offering a cure.
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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, Guangxi, China
Conditions
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