Puchkov Pavel A, Maslov Michael A
Lomonosov Institute of Fine Chemical Technologies, MIREA-Russian Technological University, Vernadsky Ave. 86, 119571 Moscow, Russia.
Pharmaceutics. 2021 Jun 21;13(6):920. doi: 10.3390/pharmaceutics13060920.
Gene therapy requires an effective and safe delivery vehicle for nucleic acids. In the case of non-viral vehicles, including cationic liposomes, the structure of compounds composing them determines the efficiency a lot. Currently, cationic amphiphiles are the most frequently used compounds in liposomal formulations. In their structure, which is a combination of hydrophobic and cationic domains and includes spacer groups, each component contributes to the resulting delivery efficiency. This review focuses on polycationic and disulfide amphiphiles as prospective cationic amphiphiles for gene therapy and includes a discussion of the mutual influence of structural components.
基因治疗需要一种有效且安全的核酸递送载体。就包括阳离子脂质体在内的非病毒载体而言,构成它们的化合物结构在很大程度上决定了效率。目前,阳离子两亲物是脂质体制剂中最常用的化合物。在其结构中,疏水结构域和阳离子结构域相结合并包含间隔基团,每个组分都对最终的递送效率有贡献。本综述重点关注作为基因治疗潜在阳离子两亲物的聚阳离子和二硫键两亲物,并讨论了结构组分之间的相互影响。