Mork Family Department of Chemical Engineering and Materials Science, Viterbi School of Engineering, University of Southern California, Los Angeles, California, USA.
Immunopharmacol Immunotoxicol. 2010 Jun;32(2):208-18. doi: 10.3109/08923970903420582.
Lentiviral vectors (LVs) are efficient gene delivery vehicles suitable for delivering long-term transgene expression in various cell types. Engineering LVs to have the capacity to transduce specific cell types is of great interest to advance the translation of LVs toward the clinic. Here we provide an overview of innovative approaches to target LVs to cells of the immune system. In this overview we distinguish between two types of LV targeting strategies: (i) targeting of the vectors to specific cells by LV surface modifications, and (ii) targeting at the level of transgene transcription by insertion of tissue-specific promoters to drive transgene expression. It is clear that each strategy is of enormous value but ultimately combining these approaches may help reduce the effects of off-target expression and improve the efficiency and safety of LVs for gene therapy.
慢病毒载体 (LVs) 是一种高效的基因传递载体,适用于在各种细胞类型中进行长期转基因表达。将 LVs 工程改造为能够转导特定细胞类型的能力,对于推进 LVs 在临床上的应用具有重要意义。本文概述了将 LVs 靶向免疫系统细胞的创新方法。在本文中,我们将 LVs 靶向策略分为两种类型:(i) 通过 LV 表面修饰将载体靶向特定细胞,以及 (ii) 通过插入组织特异性启动子来靶向转基因转录,从而驱动转基因表达。很明显,每种策略都具有巨大的价值,但最终结合这些方法可能有助于降低脱靶表达的影响,并提高 LVs 在基因治疗中的效率和安全性。