This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.
No eligiblity for allogeneic hematopoietic stem cell transplantation.
The treatment of erythrocyte maturation agent luspatercept cannot be financially supported.
The subjects' parents/legal guardians must be willing and able to follow the study procedures in the study protocol.
Good organs' functions.
Availability of voluntary, fully HLA-matched hematopoietic cell donors, unless recommended for inclusion by the Monitoring Committee.
HIV-1 and HIV-2 were positive, and / or HTLV-1, HTLV-2 and VSV-G antibodies were positive.
An active bacterial, viral, fungal or parasitic infection.
Contraindicated for the extraction of bone marrow under anesthesia.
Shanghai BDgene Co., Ltd.
Lead sponsor
Shanghai Children's Medical Center
Collaborator