Klimatcheva E, Rosenblatt J D, Planelles V
Departments of Medicine and Microbiology & Immunology, University of Rochester Cancer Center, NY 14642, USA.
Front Biosci. 1999 Jun 1;4:D481-96. doi: 10.2741/klimatcheva.
Gene therapy is a novel method under investigation for the treatment of genetic, metabolic and neurologic diseases, cancer and AIDS. The primary goal of gene therapy is to deliver a specific gene to a pre-determined target cell, and to direct expression of such a gene in a manner which will result in a therapeutic effect. Retroviral vectors have the ability to integrate in the host cell DNA irreversibly and therefore, are suitable vectors for permanent genetic modification of cells. Retrovirus-mediated gene transfer has been limited, however, by the inability of onco-retroviruses to productively infect non-dividing cells. Lentiviruses are unique among retroviruses because of their ability to infect target cells independently of their proliferation status. This chapter presents an up-to-date description of available lentiviral vectors, including vector design, applications to disease treatment and safety considerations. In addition, general aspects of the biology of lentiviruses with relevance to vector development will be discussed.
基因治疗是一种正在研究的用于治疗遗传、代谢和神经疾病、癌症及艾滋病的新方法。基因治疗的主要目标是将特定基因传递到预先确定的靶细胞，并以能产生治疗效果的方式指导该基因的表达。逆转录病毒载体有能力不可逆地整合到宿主细胞DNA中，因此，是用于细胞永久性基因改造的合适载体。然而，逆转录病毒介导的基因转移受到了致癌逆转录病毒不能有效感染非分裂细胞的限制。慢病毒在逆转录病毒中很独特，因为它们有能力独立于靶细胞的增殖状态感染靶细胞。本章提供了现有慢病毒载体的最新描述，包括载体设计、在疾病治疗中的应用以及安全考虑。此外，还将讨论与载体开发相关的慢病毒生物学的一般方面。
