Enormous studies have corroborated that long non-coding RNAs (lncRNAs) extensively participate in crucial physiological processes such as metabolism and immunity, and are closely related to the occurrence and development of tumors, cardiovascular diseases, nervous system disorders, nephropathy, and other diseases. The application of lncRNAs as biomarkers or intervention targets can provide new insights into the diagnosis and treatment of diseases. This paper has focused on the emerging research into lncRNAs as pharmacological targets and has reviewed the transition of lncRNAs from the role of disease coding to acting as drug candidates, including the current status and progress in preclinical research. Cutting-edge strategies for lncRNA modulation have been summarized, including the sources of lncRNA-related drugs, such as genetic technology and small-molecule compounds, and related delivery methods. The current progress of clinical trials of lncRNA-targeting drugs is also discussed. This information will form a latest updated reference for research and development of lncRNA-based drugs.

大量的研究证实，长的非编码RNA（lncRNA）广泛参与关键的生理过程，例如新陈代谢和免疫力，并且与肿瘤，心血管疾病，神经系统疾病，肾病和其他疾病的发生和发展密切相关。lncRNA作为生物标志物或干预目标的应用可以为疾病的诊断和治疗提供新的见识。本文将重点放在作为药物靶标的lncRNA的新兴研究上，并综述了lncRNA从疾病编码的角色到充当候选药物的转变，包括临床前研究的现状和进展。总结了lncRNA调节的前沿策略，包括与lncRNA相关的药物的来源，例如遗传技术和小分子化合物，以及相关的递送方法。还讨论了靶向lncRNA的药物的临床试验的最新进展。这些信息将构成基于lncRNA的药物研发的最新更新参考。