基础医学与临床 ›› 2022, Vol. 42 ›› Issue (1): 41-50.

• 特邀专题 • 上一篇    下一篇

基因治疗中的核酸药物及非病毒递送载体的研究进展

刘健   

  1. 中国医学科学院基础医学研究所 北京协和医学院基础学院
  • 收稿日期:2021-12-03 修回日期:2021-12-03 出版日期:2022-01-05 发布日期:2022-01-05
  • 通讯作者: 刘健 E-mail:liujian@ibms.pumc.edu.cn

Progress of nucleic acid drugs and non-viral carriers in gene therapy

Jian LIU   

  • Received:2021-12-03 Revised:2021-12-03 Online:2022-01-05 Published:2022-01-05
  • Contact: Jian LIU E-mail:liujian@ibms.pumc.edu.cn

摘要: 基因治疗是针对基因异常相关疾病的终极治疗技术,各种具有不同机制的核酸药物的出现为基因治疗带来了更多的可能性。但是,由于存在体内稳定性差、难以高效进入靶细胞等问题,核酸药物需要载体的帮助而进入目标细胞并到达特定的胞内位置,因此,开发安全高效的核酸递送系统是基因治疗的基石。与病毒载体相比,非病毒载体具有更高的安全性,但转染效率较低。随着纳米技术的发展,非病毒载体的效率得到了显著的提升,进入临床研究的数量逐渐增多。本文简要介绍基因治疗中的核酸药物及其递送载体,对非病毒核酸药物递送技术的瓶颈及进展做综合评述。

Abstract: Gene therapy based on nucleic acid drugs is the ultimate treatment technology for gene abnormality related diseases. However, nucleic acid drugs need the help of carriers to enter target cells and reach specific intracellular locations, therefore, the development of safe and efficient delivery systems for nucleic acid drugs is one of the fundamental technologies for gene therapy. Compared with viral vectors, non-viral vectors have higher safety, but are still facing the challenge of relative low transfection efficiency. With the advances of nanotechnology, the efficiency of non-viral carriers has been gained significantly improvements. In this paper, some representative nucleic acid-based drugs and vaccines were introduced, and the progresses of non-viral delivery carriers for uses in gene therapy have been reviewed.