Gene-Editing breakthrough: first patients treated for blood disorder
NCT ID NCT06065189
First seen May 16, 2026 · Last updated May 23, 2026 · Updated 2 times
Summary
This early-phase study tested a new gene-editing technique (base editing) on a patient's own blood stem cells to treat severe β-thalassemia, a genetic blood disorder requiring lifelong transfusions. Two children received the edited cells after chemotherapy, and researchers monitored safety, engraftment, and whether transfusions could be reduced or stopped. The goal is to see if this one-time treatment can control the disease long-term.
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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, 201102, China
Conditions
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