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首页> 外文期刊>International Journal of Pharmaceutics >From rationally designed polymeric and peptidic systems to sophisticated gene delivery nano-vectors
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From rationally designed polymeric and peptidic systems to sophisticated gene delivery nano-vectors

机译：从合理设计的聚合物和肽类系统到复杂的基因传递纳米载体

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摘要

Lack of safe, efficient and controllable methods for delivering therapeutic genes appears to be the most important factor preventing human gene therapy. Safety issues encountered with viral vectors have prompted substantial attention to in vivo investigations with non-viral vectors throughout the past decade. However, developing non-viral vectors with effectiveness comparable to viral ones has been a challenge. The strategy of designing multifunctional synthetic carriers targeting several extracellular and intracellular barriers in the gene transfer pathway has emerged as a promising approach to improving the efficacy of gene delivery systems. This review will explain how sophisticated synthetic vectors can be created by combining conventional polycationic vectors such as polyethylenimine and basic amino acid peptides with additional polymers and peptides that are designed to overcome potential barriers to the gene delivery process.
机译：缺乏用于递送治疗性基因的安全，有效和可控制的方法似乎是阻止人类基因治疗的最重要因素。在过去的十年中，病毒载体遇到的安全性问题已引起人们对非病毒载体体内研究的广泛关注。但是，开发具有与病毒相当的功效的非病毒载体一直是一个挑战。设计针对基因转移途径中多个细胞外和细胞内屏障的多功能合成载体的策略已经作为提高基因递送系统功效的有前途的方法出现。这篇综述将解释如何通过将常规的聚阳离子载体（如聚乙烯亚胺和碱性氨基酸肽）与旨在克服基因传递过程中潜在障碍的其他聚合物和肽相结合来创建复杂的合成载体。

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