小干扰RNA
医学
食品药品监督管理局
药物输送
临床试验
药理学
核糖核酸
生物
纳米技术
病理
基因
生物化学
材料科学
作者
Fan Guo,Yan Li,Wenjun Yu,Yuanlei Fu,Jing Zhang,Haiqiang Cao
标识
DOI:10.1021/acs.molpharmaceut.3c01158
摘要
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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