Gene-Edited cells: 15-Year watch for blood disorder breakthrough
NCT ID NCT06685536
Summary
This study tracks patients for up to 15 years after they received an experimental gene therapy for beta-thalassemia, a serious inherited blood disorder. Researchers want to understand the long-term safety and effectiveness of the treatment, which uses the patient's own genetically modified stem cells. The study follows 5 participants who received the therapy in an earlier trial, monitoring for side effects and whether they remain free from needing blood transfusions.
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Contacts and locations
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Locations
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The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China
Conditions
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