Weng Yuhua, Huang Qianqian, Li Chunhui, Yang Yongfeng, Wang Xiaoxia, Yu Jie, Huang Yuanyu, Liang Xing-Jie
Advanced Research Institute of Multidisciplinary Science, School of Life Science, Institute of Engineering Medicine, Key Laboratory of Molecular Medicine and Biotherapy, Beijing Institute of Technology, Beijing 100081, P.R. China.
Chinese Academy of Sciences (CAS) Key Laboratory for Biomedical Effects of Nanomaterials and Nanosafety, CAS Center for Excellence in Nanoscience, National Center for Nanoscience and Technology of China, Beijing 100190, P.R. China; University of Chinese Academy of Sciences, Beijing 100049, P.R. China.
Mol Ther Nucleic Acids. 2020 Mar 6;19:581-601. doi: 10.1016/j.omtn.2019.12.004. Epub 2019 Dec 17.
Due to a series of systemic and intracellular obstacles in nucleic acid (NA) therapy, including fast degradation in blood, renal clearance, poor cellular uptake, and inefficient endosomal escape, NAs may need delivery methods to transport to the cell nucleus or cytosol to be effective. Advanced nanoscale biotechnology-associated strategies, such as controlling the particle size, charge, drug loading, response to environmental signals, or other physical/chemical properties of delivery carriers, have provided great help for the in vivo and in vitro delivery of NA therapeutics. In this review, we introduce the characteristics of different NA modalities and illustrate how advanced nanoscale biotechnology assists NA therapy. The specific features and challenges of various nanocarriers in clinical and preclinical studies are summarized and discussed. With the help of advanced nanoscale biotechnology, some of the major barriers to the development of NA therapy will eventually be overcome in the near future.
由于核酸（NA）治疗中存在一系列系统性和细胞内障碍，包括在血液中快速降解、经肾脏清除、细胞摄取不佳以及内体逃逸效率低下，核酸可能需要递送方法才能转运至细胞核或细胞质中以发挥作用。先进的纳米生物技术相关策略，如控制递送载体的粒径、电荷、药物负载、对环境信号的响应或其他物理/化学性质，为核酸治疗药物的体内和体外递送提供了极大帮助。在本综述中，我们介绍了不同核酸形式的特点，并阐述了先进的纳米生物技术如何辅助核酸治疗。总结并讨论了各种纳米载体在临床和临床前研究中的具体特征及挑战。借助先进的纳米生物技术，核酸治疗发展的一些主要障碍最终将在不久的将来被克服。
