β-globin Restored Autologous HSC in β-thalassemia Major Patients

Inclusion Criteria: * 5-15 years old. Clinically diagnosed as β-thalassemia major with IVS-654 gene mutation phenotype; * Subjects or at least one legal guardian/agent understand and voluntarily sign informed consent. * Subjects with no affection with EBV, HIV, CMV, TP, HAV, HBV and HCV. * Subjects body condition eligible for autologous stem cell transplant. Exclusion Criteria: \- Subjects acceptable for allogeneic hematopoietic stem cell transplantation and have an available fully matched related donor. Active bacterial, viral, or fungal infection. Treated with erythropoietin prior 3 months. Immediate family member with any known hematological tumor. Subjects with severe psychiatric disorders to be unable to cooperate. Recently diagnosed as malaria. History of complex autoimmune disease. Persistent aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin value \>3 x the upper limit of normal (ULN). Subjects with severe heart, lung and kidney diseases. With serious iron overload. Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician or Investigator. Subjects who are receiving treatment from another clinical study, or have received another gene therapy. Subjects or guardians had resisted the guidance of the attending doctor. Subjects whom the investigators do not consider appropriate for participating in this clinical study.