Gene therapy aims to free thalassemia patients from lifelong blood transfusions
NCT ID NCT07489196
First seen Mar 27, 2026 · Last updated May 24, 2026 · Updated 8 times
Summary
This study tests a one-time gene therapy called CS-101 in 20 people aged 12 to 35 with severe β-thalassemia major who need regular blood transfusions. The goal is to see if the treatment is safe and can help patients produce enough healthy hemoglobin to stop needing transfusions for at least 12 months. Participants will be monitored for side effects and blood cell recovery after the infusion.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Conditions
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