One-Shot CRISPR treatment aims to free patients from lifelong blood transfusions

NCT ID NCT05577312

Summary

This early-stage study is testing a single infusion of a patient's own genetically modified stem cells to treat severe beta-thalassemia. The therapy uses CRISPR gene editing technology with the goal of helping the body produce healthy red blood cells. If successful, it could reduce or eliminate the need for regular blood transfusions in people with this serious inherited blood disorder.

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Contacts and locations

Locations

  • Chinese Academy of Medical Sciences

    Tianjin, Tianjin Municipality, China

  • Nanfang Hospital, Southern Medical University

    Guangzhou, Guangdong, 510510, China

  • The First Affiliated Hospital of Guangxi Medical University

    Nanning, Guangxi, 530021, China

  • Xiangya Hospital of Central South University

    Changsha, Hunan, 510510, China

Conditions

Explore the condition pages connected to this study.