Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified

Inclusion Criteria: * Subjects must be 8 years of age or older. * Subjects or their parents/legal guardians must be able to understand and voluntarily sign an informed consent form. * Subjects must have a confirmed diagnosis of ß-thalassemia major and ≥100 mL/kg/year of pRBCs or ≥ 8 transfusions of pRBCs per year over a minimum of two years prior to entry onto the study. * Subjects must be in clinically stable condition and eligible for hematopoietic stem cell transplantation. * Subjects must satisfy Karnofsky index ≥80% for adults or Lansky index ≥70% for children. * Subjects must have survival expectancy of greater than 6 months. * Subjects must have been treated and followed up for at least the past 2 years in specialized institutions where they have comprehensive assessment of the disease（including psychiatric assessment），and detailed medical materials at least the past 2years so as to self-contrast before and after treatment. * Subjects must discontinue treatment of hydroxyurea, 5-azoside or cytarabine at least three months prior to entry onto the study. Exclusion Criteria: * Having an HLA-matched donor(sibling or of a suitable 10/10 matched unrelated donor). * Positivity for HIV (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA (or negative HCV RNA but on antiviral treatment). * Contraindication to anesthesia for bone marrow collection. * Severe, bacterial, active viral, or fungal infection, etc. * The history of malignant tumor. * The white blood cell (WBC) count \<3000/uL and/or platelet count \<100,000/uL exclude hypersplenism factor. * Family history of familial cancer syndromes (including but not limited to Hereditary breast and ovarian syndrome, hereditary non-polyp colorectal cancer syndrome, familial adenomatous polyposis). * Previous allogeneic bone marrow transplantation. * The history of psychosis and any psychiatric disorder. * Active substance abuse, drug or alcohol abuse recently. * The history of complex allo-immunization which could cause difficulty administering transfusions. * Female adults who are pregnant , breast feeding or lack of effective contraception. * History of major organ damage including: Severe cerebrovascular disease or cognitive sequelae, including hemiplegia. Severe liver disease with alanine transaminase (ALT) \>3 upper limit of normal. Severe liver cirrhosis or fibrosis on liver biopsy. Heart disease with ejection fraction\<25% or T2\* \<10 ms by magnetic resonance imaging (MRI). Kidney disease with creatinine clearance \<30% normal value. Lung disease, including pulmonary fibrosis, pulmonary arterial hypertension or pulmonary function tests below standard (i.e., pO2\<90 mmHg and/or carbon dioxide diffusion coefficient\<50%). Endocrine disorder including insulin dependent diabetes mellitus, Hyperthyroidism or deficiency, Hyperparathyroidism or deficiency. * Participation in another clinical study within 30 days of screening. * Subjects with severe iron overload determined by the researchers. * Any other situation that unsuitably undergoing hematopoietic stem cell transplantation determined by the physicians or researchers. * Presence of chromosomal abnormalities by bone marrow detected.