Gene-Editing breakthrough offers hope for blood disorder patients
NCT ID NCT06065189
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage study tested a new gene-edited stem cell transplant in 2 children with severe β-thalassemia, a serious blood disorder requiring lifelong transfusions. The treatment aims to help the body produce healthy red blood cells and reduce or stop the need for transfusions. Researchers monitored safety, engraftment, and blood counts to see if the approach is feasible.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102, China
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Other studies related to the condition(s) this trial covers.
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