Hardee Cinnamon L, Arévalo-Soliz Lirio Milenka, Hornstein Benjamin D, Zechiedrich Lynn
Interdepartmental Program in Integrative Molecular and Biomedical Sciences, Baylor College of Medicine, Houston, TX 77030, USA.
Department of Molecular Virology and Microbiology, Baylor College of Medicine, Houston, TX 77030, USA.
Genes (Basel). 2017 Feb 10;8(2):65. doi: 10.3390/genes8020065.
Uses of viral vectors have thus far eclipsed uses of non-viral vectors for gene therapy delivery in the clinic. Viral vectors, however, have certain issues involving genome integration, the inability to be delivered repeatedly, and possible host rejection. Fortunately, development of non-viral DNA vectors has progressed steadily, especially in plasmid vector length reduction, now allowing these tools to fill in specifically where viral or other non-viral vectors may not be the best options. In this review, we examine the improvements made to non-viral DNA gene therapy vectors, highlight opportunities for their further development, address therapeutic needs for which their use is the logical choice, and discuss their future expansion into the clinic.
到目前为止，在临床基因治疗递送中，病毒载体的应用已使非病毒载体的应用黯然失色。然而，病毒载体存在一些问题，包括基因组整合、无法重复递送以及可能的宿主排斥反应。幸运的是，非病毒DNA载体的开发一直在稳步推进，特别是在质粒载体长度缩短方面，现在这些工具能够在病毒载体或其他非病毒载体可能不是最佳选择的特定领域发挥作用。在本综述中，我们研究了非病毒DNA基因治疗载体所取得的进展，强调其进一步发展的机会，探讨选择其作为治疗手段的合理依据的治疗需求，并讨论其未来在临床中的扩展应用。
