β-地中海贫血基因治疗的研究进展

Progression of gene therapy for β-thalassemia

β-地中海贫血基因治疗的策略和方法,根据靶向的不同可以分为3类:导入正常的β-珠蛋白基因、导入正常的γ-珠蛋白基因和抑制α-珠蛋白基因的表达。β-地中海贫血动物模型的发展,对疾病基因治疗的进步有重要意义。基因治疗载体系统是关系到基因治疗成败的重要因素之一,其中慢病毒载体的采用是一重要进展。

The strategies and methods of gene therapy for β-thalassemia can be divided into 3 categories：transfer of normal β-globin gene,transfer of normal γ-globin gene,and inhibition of α-globin gene expression.The gene therapy of β-thalassemia cannot make progress without the development of suitable animal models of the disease.The vector system for gene therapy is one of important factors which contribute to the success of gene therapy.The use of lentiviral vector is a key step moved forward in gene therapy for β-thalassemia.