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.

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小干扰RNA递送的市场及临床阶段最新进展

小干扰 RNA (siRNA) 是一种很有前途的治疗策略，五种 siRNA 药物已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 临床应用开发的一个重要里程碑。批准的siRNA药物可以有效地将siRNA递送至肝脏并治疗肝脏相关疾病。目前，研究人员已开发出多种递送平台，用于将siRNA转运至脑、肺、肌肉等不同组织，并且大量siRNA药物正在进行临床试验。在此，总结了这些递送技术和临床应用的最新进展，本综述简要概述了将 siRNA 递送至肝组织和肝外组织所采用的策略。