小干扰RNA递送系统和化学修饰的研究进展
Research progress of small interfering RNA delivery system and chemical modification
赵晓东 1陈华英 2陈涛 1张燕媚 3李华2
作者信息
- 1. 中国医药工业研究总院,上海 201203;上海益诺思生物技术股份有限公司,上海 201203
- 2. 上海益诺思生物技术股份有限公司,上海 201203
- 3. 中国医药工业研究总院,上海 201203;深圳市益诺思生物医药安全评价研究院有限公司,广东深圳 518000
- 折叠
摘要
2018年美国FDA批准首个小干扰RNA(small interfering RNA,siRNA)药物patisiran,标志着RNA干扰疗法进入一个新时代.siRNA凭借独特的成药优势,近些年发展迅速.目前已有5款siRNA药物获FDA批准上市.然而,其在治疗应用中的靶向性和疗效方面仍存诸多问题.为开发出具有更多潜在价值的新产品,有必要深入探讨siRNA药物研发现状.该文综述siRNA在成药方面的优势与阻碍,重点讨论siRNA药物的化学修饰策略和递送系统的研究进展.以期为后续研究提供参考.
Abstract
In 2018,the U.S.FDA approved the first small interfering RNA(siRNA)drug Patisiran,marking a brand-new era for RNA interference therapy.siRNA has developed rapidly in recent years due to its unique drug-forming advantages.Five siRNA drugs have been approved for marketing by the FDA.However,there are still many problems in targeting and efficacy in therapeutic applications.In order to develop new products with more potential value,it is necessary to deeply explore the current status of siRNA drug discovery and development.This article reviewed the advantages and hindrances of siRNA in drug discovery,focused on the research progress of chemical modification strategies and delivery systems of siRNA drugs,in order to provide a reference for the subsequent research.
关键词
小干扰RNA/RNA干扰/递送系统/化学修饰Key words
small interfering RNA/RNA interference/delivery system/chemical modification引用本文复制引用
出版年
2024
NETL
NSTL
万方数据