Zufferey R, Nagy D, Mandel R J, Naldini L, Trono D
Salk Institute, La Jolla, CA 92037-1099, USA.
Nat Biotechnol. 1997 Sep;15(9):871-5. doi: 10.1038/nbt0997-871.
Retroviral vectors derived from lentiviruses such as HIV-1 are promising tools for human gene therapy because they mediate the in vivo delivery and long-term expression of transgenes in nondividing tissues. We describe an HIV vector system in which the virulence genes env, vif, vpr, vpu, and nef have been deleted. This multiply attenuated vector conserved the ability to transduce growth-arrested cells and monocyte-derived macrophages in culture, and could efficiently deliver genes in vivo into adult neurons. These data demonstrate the potential of lentiviral vectors in human gene therapy.
源自诸如HIV-1等慢病毒的逆转录病毒载体是人类基因治疗的有前景的工具，因为它们介导转基因在非分裂组织中的体内递送和长期表达。我们描述了一种HIV载体系统，其中毒力基因env、vif、vpr、vpu和nef已被删除。这种多重减毒载体保留了在培养中转导生长停滞细胞和单核细胞衍生巨噬细胞的能力，并且能够在体内有效地将基因递送至成年神经元。这些数据证明了慢病毒载体在人类基因治疗中的潜力。
