What can we learn from virus in designing nonviral gene vectors
What can we learn from virus in designing nonviral gene vectors
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DOI：
10.1007/s10118-011-1047-5
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发表时间：
2011-04
影响因子：
4.3
通讯作者：
Chunxiao Xu;M. Sui;Jianbin Tang;Youqing Shen
中科院分区：
文献类型：
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作者：
Chunxiao Xu;M. Sui;Jianbin Tang;Youqing Shen
基因治疗已经成为一种潜在的新方法，通过将治疗基因递送到靶向病变组织来治疗遗传性疾病。然而，其临床应用受到基因递送系统的阻碍。病毒载体在递送和表达其携带的基因方面非常有效，但它们在临床使用中存在安全性问题。虽然非病毒载体经过精心的材料设计，具有很低的风险和安全性，但其基因转录效率太低，不能应用于临床。因此，合理设计模拟病毒载体的非病毒载体将是打破这一瓶颈的一种方法。本文综述了病毒/非病毒基因载体如何在血液循环、细胞摄取、内体逃逸、核输入和基因转录等方面超越这些生物屏障。
Gene therapy has emerged as a potential new approach to treat genetic disorders by delivering therapeutic genes to target diseased tissues. However, its clinical use has been impeded by gene delivery systems. The viral vectors are very efficient in delivering and expressing their carried genes, but they have safety issues in clinical use. While nonviral vectors are much safer with very low risks after careful material design, but their gene transcription efficiency is too low to be clinically used. Thus, rational design of nonviral vectors mimicking the viral vectors would be a way to break this bottleneck. This review compares side-by-side how viral/nonviral gene vectors transcend these biological barriers in terms of blood circulation, cellular uptake, endosome escape, nucleus import and gene transcription.