Study Summary
The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT children with lifelong supportive therapy and healthy children.
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Request More InfoInterventions
Gene therapyGENETIC
Autologous edited hematopoietic stem cell transplantation
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Regenerative Medicine Center | Tianjin | Tianjin Municipality | China |