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市场和临床阶段的小干扰 RNA 递送的最新进展。

Recent Progress of Small Interfering RNA Delivery on the Market and Clinical Stage.

机构信息

School of Pharmacy, Binzhou Medical University, Shandong 264003, China.

Yantai Key Laboratory of Nanomedicine & Advanced Preparations, Yantai Institute of Materia Medica, Shandong 264000, China.

出版信息

Mol Pharm. 2024 May 6;21(5):2081-2096. doi: 10.1021/acs.molpharmaceut.3c01158. Epub 2024 Apr 17.

Abstract

Small interfering RNAs (siRNAs) are promising therapeutic strategies, and five siRNA drugs have been approved by the Food and Drug Administration (FDA) and the European Commission (EC). This marks a significant milestone in the development of siRNA for clinical applications. The approved siRNA agents can effectively deliver siRNAs to the liver and treat liver-related diseases. Currently, researchers have developed diverse delivery platforms for transporting siRNAs to different tissues such as the brain, lung, muscle, and others, and a large number of siRNA drugs are undergoing clinical trials. Here, these delivery technologies and the latest advancements in clinical applications are summarized, and this Review provides a concise overview of the strategies employed for siRNA delivery to both hepatic and extrahepatic tissues.

摘要

小干扰 RNA(siRNA)是一种很有前途的治疗策略,已有 5 种 siRNA 药物获得美国食品和药物管理局(FDA)和欧盟委员会(EC)的批准。这标志着 siRNA 临床应用发展的一个重要里程碑。已批准的 siRNA 药物可以有效地将 siRNA 递送到肝脏并治疗肝脏相关疾病。目前,研究人员已经开发了多种输送平台,将 siRNA 输送到不同的组织,如脑、肺、肌肉等,并且大量的 siRNA 药物正在进行临床试验。在这里,总结了这些输送技术和临床应用的最新进展,本综述简要概述了用于将 siRNA 递送到肝内和肝外组织的策略。

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