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非病毒载体阳离子基因递送试剂的研究进展

Progress of cationic gene delivery reagents for non-viral vector.

作者信息

Ma Kai, Mi Chun-Liu, Cao Xiang-Xiang, Wang Tian-Yun

机构信息

Department of Pharmacy, Xinxiang Medical University, Xinxiang, 453003, Henan, China.

International Joint Research Laboratory for Recombinant Pharmaceutical Protein Expression System of Henan, Xinxiang Medical University, Xinxiang, 453003, Henan, China.

出版信息

Appl Microbiol Biotechnol. 2021 Jan;105(2):525-538. doi: 10.1007/s00253-020-11028-6. Epub 2021 Jan 4.

Abstract

Gene delivery systems play a vital role in gene therapy and recombinant protein production. The advantages of using gene delivery reagents for non-viral vector include the capacity to accommodate a large packaging load and their low or absent immunogenicity. Furthermore, they are easy to produce at a large scale and preserve. Gene delivery reagents for non-viral vector are commonly used for transfecting a variety of cells and tissues. It is mainly composed of liposomes and non-liposome cationic polymers. According to the different head structures used, the non-viral cationic transfection reagents include a quaternary ammonium salt, amine, amino acid or polypeptide, guanidine salt, and a heterocyclic ring. This article summarizes these approaches and developments of types and components of transfection reagents and optimization of gene delivery. The optimization of mammalian cell transient recombinant protein expression system and cationic reagents for clinical or clinical trials are also discussed.

摘要

基因递送系统在基因治疗和重组蛋白生产中发挥着至关重要的作用。使用基因递送试剂作为非病毒载体的优势包括能够容纳大量包装负载以及低免疫原性或无免疫原性。此外,它们易于大规模生产和保存。非病毒载体的基因递送试剂通常用于转染多种细胞和组织。它主要由脂质体和非脂质体阳离子聚合物组成。根据所使用的不同头部结构,非病毒阳离子转染试剂包括季铵盐、胺、氨基酸或多肽、胍盐以及杂环。本文总结了转染试剂的类型、成分以及基因递送优化的这些方法和进展。还讨论了哺乳动物细胞瞬时重组蛋白表达系统和用于临床或临床试验的阳离子试剂的优化。

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