反义寡核苷酸递送方法研究进展

如何将反义寡核苷酸 (AS ODNs)有效递送进入细胞是反义核酸领域面临的一大难题。近年来 ,出现了多种寡核苷酸 (ODNs)的递送方法。在培养细胞中 ,使用的递送方法包括阳离子载体包裹、特异受体的配体导向、ODNs偶联修饰、细胞膜辅助穿透以及利用逆转录病毒载体转染等 ,其应用有效增强了AS ODNs的作用效果 ,大幅度降低了AS ODNs的使用浓度 ;在体内 ,由于临床使用裸露AS ODNs连续给药能达到一定的反义效果 ,而使递送方法的研究和应用尚处于初步尝试和探索之中 ,迄今报道的递送方法有脂类和非脂类两类。

Progress of Delivery Systems for Antisense Oligonucleotides

Efficient delivery of antisense oligodeoxyribonucleotides(AS-ODNs) into cells is one of the major problems in the field of antisense nucleic acid.Over the past few years,a range of delivery systems have been developed.In vitro,the useful delivery systems include cationic vector encapsulation,ligands targeting,conjugated ODNs,penetration into cells by acting on the plasma membrane,retroviral vector transfection,etc.And they have been found to obviously increase the efficacy of AS\|ODNs and strongly decrease concentrations of AS\|ODNs.In vivo,the antisense effects can be achieved by sustained administration of naked ODNs,so far only limited information about the delivery systems in vivo is available.And the reported delivery systems are classified into lipidic and non-lipidic.;