Gene therapy could free blood disease patients from transfusions
NCT ID NCT07489196
First seen Mar 27, 2026 · Last updated May 12, 2026 · Updated 7 times
Summary
This study tests a single dose of CS-101, a gene therapy, in 20 people aged 12 to 35 with β-thalassemia major, a severe blood disorder requiring regular transfusions. The goal is to see if it 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 treatment.
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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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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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