Gene therapy breakthrough offers hope for blood disorder patients
NCT ID NCT06291961
First seen Feb 14, 2026 · Last updated May 23, 2026 · Updated 13 times
Summary
This early-stage trial tested a single dose of CS-101, a gene therapy, in 9 people with beta-thalassemia major, a severe blood disorder requiring lifelong transfusions. The main goals were to check safety and see if the treatment helps the body produce healthy red blood cells. While not a cure, it aims to reduce or eliminate the need for regular blood transfusions.
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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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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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