Chen Yuanyuan, Li Zhaojun, Chen Xiaoguang, Zhang Sen
State Key Laboratory of Bioactive Substances and Functions of Natural Medicines, Institute of Materia Medica, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing 100050, China.
Acta Pharm Sin B. 2021 Feb;11(2):340-354. doi: 10.1016/j.apsb.2020.10.001. Epub 2020 Oct 10.
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.
大量研究证实,长链非编码RNA(lncRNAs)广泛参与代谢和免疫等关键生理过程,并与肿瘤、心血管疾病、神经系统疾病、肾病及其他疾病的发生发展密切相关。将lncRNAs用作生物标志物或干预靶点可为疾病的诊断和治疗提供新的思路。本文聚焦于将lncRNAs作为药理学靶点的新兴研究,并综述了lncRNAs从疾病编码角色向作为候选药物角色的转变,包括临床前研究的现状和进展。总结了lncRNA调控的前沿策略,包括lncRNA相关药物的来源,如基因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药物的临床试验的当前进展。这些信息将为基于lncRNA的药物研发形成最新的参考资料。