One-Shot gene therapy aims to free patients from lifelong blood transfusions
NCT ID NCT07489196
Summary
This study is testing a single injection of a patient's own genetically modified cells to treat severe beta-thalassemia. The goal is to see if this one-time treatment is safe and can help patients produce enough of their own healthy hemoglobin to stop needing regular blood transfusions. The trial will enroll about 20 participants aged 12 to 35 who currently depend on transfusions to survive.
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Contacts and locations
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Study contacts
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Phone: •••-•••-•••• Email: •••••@•••••
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Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
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Ruijin Hospital Shanghai JiaoTong University School of Medicine
Shanghai, Shanghai Municipality, China
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The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China
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Conditions
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