Clinical Research Directory

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-transplantation Exclusion 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.