慢病毒载体与造血干细胞介导的基因治疗

慢病毒载体不仅能感染造血干细胞 (HSCs) ,使携带的目的基因整合至HSCs基因组内 ,且能利用病毒携带的调控元件 ,使目的基因随HSCs细胞特异性表达 ,因此是一种有效的感染HSCs和进行基因治疗的工具。主要对慢病毒载体基因组特点、改建过程、慢病毒对干细胞的感染能力、慢病毒携带的目的基因在HSCs内的表达及调控等方面做了简要的综述

Lentivirus Vectors and Gene Therapy by Using Hematopoietic Stem Cells

Lentivirus vector is an effective tool for the studies of gene therapy. The vector can not only effectively infect the target cells, hematopoietic stem cells (HSCs), but also make the genes carried by lentivirus vector integrate into genome of HSCs. In addition, foreign genes can express specially in HSCs by using the regulation elements in the lentivirus vector. In this article, we breifly reviewed the genomic characteristics of lentivirus vector, the development of the vector constructs, the infective ability to HSCs, the expression and regulation of genes carried by lentivirus vectors.