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.
The tumor genes chip detection results about acute leukemia and myeloid tumor gene mutations (panel) showed no abnormality.
There were candidates for HLA gene semi-compatible hematopoietic stem cell transplantation.
No eligiblity for allogeneic hematopoietic stem cell transplantation.
The treatment of erythrocyte maturation agent luspatercept cannot be financially supported.
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
Ruijin Hospital
Collaborator