This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients
8-16 years old. Subject and/or subject's legal guardian fully understand and voluntarily sign informed consent;
Clinically diagnosed as transfusion-dependent β-thalassemia major;
With sufficient RBC infusion, subjects must maintain hemoglobin ≥9g/dL, serum ferritin threshold ≤ 3000 ng/mL and the liver iron overload mild or absent for at least 3 months before mobilization of hematopoietic stem cell;
Follow the arrangements for treatment and regular medical checks within two years post-transplantation
The physical condition does not meet the requirements for hematopoietic stem cell mobilization and transplantation myeloablation;
Received gene therapy and allogeneic HSCT in the past.
Have an available HLA matched donor.
Enrolling in another clinical trial.