Gene therapy breakthrough offers hope for blood disorder patients
NCT ID NCT06291961
First seen Feb 14, 2026 · Last updated May 15, 2026 · Updated 11 times
Summary
This early-stage study tested a single dose of CS-101 gene therapy in 9 people with beta-thalassemia major, a severe blood disorder requiring lifelong transfusions. The goal was to see if the treatment is safe and can help the body produce healthy hemoglobin. While promising, patients may still need ongoing care, so this is not a cure.
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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
Conditions
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