基于RNA的治疗法在癌症、传染病和代谢性疾病等多个医疗应用中显示出巨大的潜力。近期mRNA疫苗在对抗COVID-19疫情方面的成功突显了RNA药物的医疗价值。然而，实现RNA药物全部潜力的一个主要挑战是以有针对性的方式将RNA输送至特定器官和组织，这对于实现治疗效果、减少副作用和提高整体治疗效果至关重要。已经尝试了多种方法进行针对性输送，然而缺乏明确的指导和共性解释阻碍了RNA药物的临床应用。在本综述中，我们概述了针对性RNA输送系统的作用机制，并总结了影响RNA药物针对性输送的四个关键因素。这些因素包括载体材料类别、载体的化学结构、给药途径和RNA载体的物理化学性质，它们显著地影响特定器官/组织的热带性。此外，我们还概述了目前正在临床试验中的主要基于RNA的药物，重点介绍了它们的设计策略和组织热带应用。本综述将有助于理解针对性输送系统的原理和机制，加速未来针对不同疾病的RNA药物的研发。© 作者。

RNA-based therapeutics have shown great promise in various medical applications, including cancers, infectious diseases, and metabolic diseases. The recent success of mRNA vaccines for combating the COVID-19 pandemic has highlighted the medical value of RNA drugs. However, one of the major challenges in realizing the full potential of RNA drugs is to deliver RNA into specific organs and tissues in a targeted manner, which is crucial for achieving therapeutic efficacy, reducing side effects, and enhancing overall treatment efficacy. Numerous attempts have been made to pursue targeting, nonetheless, the lack of clear guideline and commonality elucidation has hindered the clinical translation of RNA drugs. In this review, we outline the mechanisms of action for targeted RNA delivery systems and summarize four key factors that influence the targeting delivery of RNA drugs. These factors include the category of vector materials, chemical structures of vectors, administration routes, and physicochemical properties of RNA vectors, and they all notably contribute to specific organ/tissue tropism. Furthermore, we provide an overview of the main RNA-based drugs that are currently in clinical trials, highlighting their design strategies and tissue tropism applications. This review will aid to understand the principles and mechanisms of targeted delivery systems, accelerating the development of future RNA drugs for different diseases.© The author(s).