The growing understanding of RNA functions and their crucial roles in diseases promotes the application of various RNAs to selectively function on hitherto “undruggable” proteins, transcripts and genes, thus potentially broadening the therapeutic targets. Several RNA-based medications have been approved for clinical use, while others are still under investigation or preclinical trials. Various techniques have been explored to promote RNA intracellular trafficking and metabolic stability, despite significant challenges in developing RNA-based therapeutics. In this review, the mechanisms of action, challenges, solutions, and clinical application of RNA-based therapeutics have been comprehensively summarized.

对 RNA 功能及其在疾病中的关键作用的日益了解促进了各种 RNA 的应用，以选择性地作用于迄今为止“不可药用”的蛋白质、转录物和基因，从而潜在地拓宽了治疗靶点。几种基于 RNA 的药物已被批准用于临床，而其他药物仍在研究或临床前试验中。尽管在开发基于 RNA 的疗法方面面临重大挑战，但已经探索了各种技术来促进 RNA 细胞内运输和代谢稳定性。在这篇综述中，全面总结了基于 RNA 的疗法的作用机制、挑战、解决方案和临床应用。