Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients

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

Inclusion Criteria: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-transplantationExclusion Criteria: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.Other unsuitable conditions identified by doctors.