CRISPR One-Shot aims to free patients from lifelong blood transfusions
NCT ID NCT05577312
Summary
This study is testing a one-time gene therapy called BRL-101 for people with a severe inherited blood disorder called transfusion-dependent beta-thalassemia. The therapy uses CRISPR technology to edit a patient's own blood stem cells, with the goal of reducing or eliminating their need for regular blood transfusions. The trial will enroll up to 39 participants, aged 3 to 35, to first check safety and then see how well the treatment works.
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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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