One-Shot gene therapy aims to cure devastating blood disorder
NCT ID NCT06291961
Summary
This early-stage study tested a new one-time gene therapy called CS-101 in 9 people with beta-thalassemia major, a severe inherited blood disease. Doctors collected each patient's own blood stem cells, used gene editing to fix the underlying genetic cause, and then gave them back. The main goal was to check if this treatment is safe and to see early signs of whether it can help the body make healthy blood again, potentially curing the disease.
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Contacts and locations
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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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