克服基因传递障碍：非病毒载体的生理学考量
Overcoming Gene-Delivery Hurdles: Physiological Considerations for Nonviral Vectors.
作者信息
Hill Andrew B, Chen Mingfu, Chen Chih-Kuang, Pfeifer Blaine A, Jones Charles H
机构信息
Abcombi Biosciences Inc, Buffalo, NY, USA; McKetta Department of Chemical Engineering, The University of Texas at Austin, Austin, TX, USA.
Department of Chemical and Biological Engineering, University at Buffalo, The State University of New York, Buffalo, BY, USA.
出版信息
Trends Biotechnol. 2016 Feb;34(2):91-105. doi: 10.1016/j.tibtech.2015.11.004. Epub 2015 Dec 23.
With the use of contemporary tools and techniques, it has become possible to more precisely tune the biochemical mechanisms associated with using nonviral vectors for gene delivery. Consequently, nonviral vectors can incorporate numerous vector compositions and types of genetic cargo to develop diverse genetic therapies. Despite these advantages, gene-delivery strategies using nonviral vectors have poorly translated into clinical success due to preclinical experimental design considerations that inadequately predict therapeutic efficacy. Furthermore, the manufacturing and distribution processes are critical considerations for clinical application that should be considered when developing therapeutic platforms. In this review, we evaluate potential avenues towards improving the transition of gene-delivery technologies from in vitro assessment to human clinical therapy.
随着当代工具和技术的应用，更加精确地调节与使用非病毒载体进行基因递送相关的生化机制已成为可能。因此，非病毒载体可以包含多种载体组成和基因载荷类型，以开发多样化的基因疗法。尽管具有这些优势，但由于临床前实验设计考虑因素未能充分预测治疗效果，使用非病毒载体的基因递送策略在临床应用中取得的成功有限。此外，制造和分发过程是临床应用的关键考虑因素，在开发治疗平台时应予以考虑。在本综述中，我们评估了改善基因递送技术从体外评估向人类临床治疗转化的潜在途径。
Zotero 插件