Gene editing breakthrough: One-Time infusion may free patients from lifelong blood transfusions
NCT ID NCT05577312
First seen Jan 18, 2026 · Last updated May 20, 2026 · Updated 20 times
Summary
This study tests a single-dose gene therapy (BRL-101) for people with transfusion-dependent beta-thalassemia, a severe blood disorder requiring regular transfusions. The treatment uses the patient's own blood stem cells, modified with CRISPR-Cas9, to help the body produce healthy red blood cells. The goal is to reduce or eliminate the need for transfusions. The study involves 39 participants aged 3-35 and will monitor safety and effectiveness for one year.
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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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Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, China
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Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510510, China
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The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, 530021, China
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Xiangya Hospital of Central South University
Changsha, Hunan, 510510, China
Conditions
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