多聚物基因递送系统：困境与对策。
Polycations for Gene Delivery: Dilemmas and Solutions.
机构信息
Key Laboratory of Polymer Ecomaterials, Changchun Institute of Applied Chemistry , Chinese Academy of Sciences , Changchun 130022 , P. R. China.
University of Science and Technology of China , Hefei 230026 , P. R. China.
出版信息
Bioconjug Chem. 2019 Feb 20;30(2):338-349. doi: 10.1021/acs.bioconjchem.8b00688. Epub 2018 Nov 12.
Gene therapy has been a promising strategy for treating numerous gene-associated human diseases by altering specific gene expressions in pathological cells. Application of nonviral gene delivery is hindered by various dilemmas encountered in systemic gene therapy. Therefore, solutions must be established to address the unique requirements of gene-based treatment of diseases. This review will particularly highlight the dilemmas in polycation-based gene therapy by systemic treatment. Several promising strategies, which are expected to overcome these challenges, will be briefly reviewed. This review will also explore the development of polycation-based gene delivery systems for clinical applications.
基因治疗通过改变病理细胞中特定基因的表达，为治疗许多与基因相关的人类疾病提供了一种很有前途的策略。非病毒基因传递的应用受到系统基因治疗中遇到的各种困境的阻碍。因此，必须建立解决方法来满足基于基因治疗疾病的独特需求。本综述将特别强调系统治疗中基于聚阳离子的基因治疗的困境。还将简要回顾几种有望克服这些挑战的有前途的策略。本综述还将探讨基于聚阳离子的基因传递系统在临床应用中的发展。
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