Inclusion Criteria:
* Patients or parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedules.
* Diagnosis of beta-thalassemia and a history of RBCs transfusions.
* Documented baseline, or pretransfusion, Hb≤7 g/dL.
* Availability of an adequate and well-documented transfusion history.
Exclusion Criteria:
* Active bacterial, viral, fungal, or parasitic infection.
* A white blood cell (WBC) counts\<3×10\^9/L, and/or platelet counts\<100×10\^9/L not related to hypersplenism.
* Uncorrected bleeding disorder.
* Presence of severe diseases that judged not compatible with the study procedures, such as severe hepatic disease, kidney disease, lung disease, and/or cardiovascular disease.
* Uncontrolled seizure disorder.
* Any evidence of severe iron overload that, in the investigator's opinion, warrants exclusion.
* Prior autologous hematopoietic stem cell transplantation.
* Prior receipt of gene therapy.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov