核酸药物是一种可在基因水平上发挥作用的核苷酸类药物，在癌症、病毒感染等疾病治疗 中具有很大的潜力。传统治疗的效果通常是短暂的，因为其目标是蛋白质而不是潜在的基因。相比之 下，核酸疗法通过基因抑制、添加、替换或编辑可以达到长期的疗效。目前应用较多的核酸药物主要包括 DNA 药物、mRNA 药物以及 siRNA 药物等。本文综述核酸药物的工作原理以及核酸药物在目前所面临的 挑战及解决办法等。

Nucleic acid drugs，as nucleotide drugs play a role on the gene level，have great potential in the treatment of some diseases such as cancer and viral infection. The therapeutic effects of traditional treatments are usually short⁃lived because they target proteins rather than underlying genes. Nucleic acid therapy，by contrast，can achieve long ⁃ term efficacy through gene suppression，addition，replacement or editing.At present，nucleic acid drugs mainly include DNA drugs，mRNA drugs and siRNA drugs. In this paper，we reviewed the working principle of these nucleic acid drugs，as well as the challenges and solutions of nucleic acid drugs in clinical application.