基因治疗作为一种诞生不久的新型治疗手段，其在面对一些难以成药的疾病治疗中展现出了巨大的潜力。近年来，伴随着RNA技术的迭代，RNA治疗成为基因治疗的重要组成部分，取得了显著进展。本文总结了RNA治疗的研究进展，包括其在疾病中的应用、临床试验结果以及未来发展方向，并探讨其在疾病治疗中的潜力与应用前景。RNA治疗能够精准调控特定的基因或RNA分子，且基于RNA的药物设计和制备过程相对简便，可根据不同疾病的治疗需求快速定制治疗方案。基于RNA分子拓展出的新型药物靶点，可以为复杂疾病提供全新的治疗视角，对未来实现精准医疗具有重要的意义。

Gene therapy emerged as a promising novel treatment for undruggable diseases. Over the past few years, RNA therapy has emerged as a pivotal subset of gene therapy, with the development of the RNA technology. Here, we summarized the cutting edge investigations of RNA therapy, with a focus on its applications in treating various diseases, the results from clinical trials, and its future prospects. RNA therapy enables the precise regulation of dysfunction or disorder genes. Given the relatively straightforward design and preparation processes of the RNA-based medicines, they can be swiftly developed into novel therapies for diverse diseases. Moreover, RNA molecules offer new avenues for treating complex diseases and are poised to play a crucial role in the realization of precision medicine in the future.