β-地中海贫血是由于β-珠蛋白链合成障碍导致β-珠蛋白生产减少的遗传性疾病。目前国内外对地中海贫血治疗分为产前诊断、规范性长期输血和去铁治疗〔1〕、HLA相合的造血干细胞移植〔2〕和脾切除术等。输血治疗存在铁负荷导致的并发症,骨髓移植则一直无法摆脱免疫排斥和移植抗宿主病的并发症,而造血干细胞具有重建个体造血系统的全能性和自我更新的能力,理论上将正常的β-珠蛋白基因导入造 β-thalassemia is a genetic disease that results in a decrease in β-globin production due to obstacles in β-globin chain synthesis. At present, the treatment of thalassemia at home and abroad is divided into prenatal diagnosis, normative long-term blood transfusion and iron therapy 〔1〕, HLA-compatible hematopoietic stem cell transplantation 〔2〕 and splenectomy. Blood transfusions are associated with iron overload. Bone marrow transplants have been unable to escape the complications of immune rejection and graft-versus-host disease, whereas hematopoietic stem cells have the capacity to reconstruct pluripotency and self-renewal of an individual’s hematopoietic system. Theoretically, - Introduction of globin genes