Research progress in antisense drug mechanism and chemical modification strategies

VernacularTitle:反义药物作用机制及化学修饰研究进展
Author: Lei LI ; Yi-xuan ZHENG ; Chun-ying MA ; Wen-hua FENG
Publication Type:Research Article
Keywords: antisense drug; antisense drug design; nucleic acid target; chemical modification; rug delivery
From: Acta Pharmaceutica Sinica 2022;57(9):2709-2719
CountryChina
Language:Chinese
Abstract: Over the past three decades, more and more antisense drugs have been approved for marketing or clinical trails. Antisense technology has become the focus of pharmaceutical research due to its unique advantages in treating diseases and strong clinical development potential. There is a big difference from traditional small molecule chemical drugs, and macromolecular protein biological drugs. Antisense drugs are a very independent drug form. Antisense drugs were initially used to treat diseases with single gene mutations, but recently they have gradually begun to be used for the treatment of common diseases. Rational antisense drug design is crucial for disease treatment based on genetics. This paper reviews the latest progress in the field of action mechanism, chemical modification and delivery strategy of antisense drugs, and analyzes the current intractable problems. It is believed that with the resolution of these problems, the research of antisense drugs can reach a new level.