One-Time gene injection aims to free patients from lifelong blood transfusions
NCT ID NCT06291961
Summary
This early-stage study tested a new, one-time gene therapy called CS-101 in 9 patients with a severe inherited blood disorder called beta-thalassemia major. The goal was to see if the treatment was safe and if it could help the patients' bodies make healthy red blood cells on their own, potentially reducing or eliminating their need for regular blood transfusions. The therapy works by editing a patient's own blood stem cells to restart production of a helpful type of hemoglobin.
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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
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
Conditions
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