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小干扰RNA递送系统和化学修饰的研究进展

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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.
Research progress of small interfering RNA delivery system and chemical modification
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.

small interfering RNARNA interferencedelivery systemchemical modification

赵晓东、陈华英、陈涛、张燕媚、李华

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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

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

小干扰RNA RNA干扰 递送系统 化学修饰

2024

世界临床药物
上海医药工业研究院

世界临床药物

CSTPCD
影响因子:0.849
ISSN:1672-9188
年,卷(期):2024.45(7)