Gene-Edited stem cells offer hope for blood disorder patients

NCT ID NCT06065189

First seen May 16, 2026 · Last updated May 16, 2026

Summary

This study tested a new treatment for β-thalassemia major, a severe blood disorder that requires lifelong blood transfusions. Two children received their own stem cells that were gene-edited in a lab to produce healthier red blood cells. The goal was to see if the treatment is safe and can reduce or eliminate the need for transfusions.

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Contacts and locations

Locations

  • Children's Hospital of Fudan University

    Shanghai, Shanghai Municipality, 201102, China

Conditions

Explore the condition pages connected to this study.