Pfützner Wolfgang
Department of Dermatology and Allergology, Philipps-University Marburg, Marburg, Germany.
Drug News Perspect. 2008 Nov;21(9):473-80. doi: 10.1358/dnp.2008.21.9.1290817.
The co-expression of genes can greatly enhance the efficiency and versatility of gene therapy applications. A variety of options exists to simultaneously express two genes in genetically modified cells. The most common approach relies on bicistronic vectors in which the genes are linked to each other by an internal ribosome entry site allowing co-translational expression of both cistrons. This review gives an overview of gene vector systems currently in use for coordinated expression of transgenes with particular focus on retroviral bicistronic vectors. The structural and functional characteristics of bicistronic vectors are discussed as well as factors influencing their efficiency such as the nature of the transgenes and the cell types in which they are expressed. Finally, the potential for clinical applications of retroviral bicistronic vectors in molecular genetics is highlighted.
基因的共表达能够极大地提高基因治疗应用的效率和通用性。在基因修饰细胞中同时表达两个基因存在多种选择。最常见的方法依赖于双顺反子载体,其中基因通过内部核糖体进入位点相互连接,允许两个顺反子共翻译表达。本综述概述了目前用于转基因协调表达的基因载体系统,特别关注逆转录病毒双顺反子载体。讨论了双顺反子载体的结构和功能特征,以及影响其效率的因素,如转基因的性质和它们所表达的细胞类型。最后,强调了逆转录病毒双顺反子载体在分子遗传学临床应用中的潜力。