MOE Key Laboratory of Gene Function and Regulation, State Key Laboratory for Biocontrol, Sun Yat-sen University, Guangzhou, 510275, China.
School of Life Science, Sun Yat-sen University, Guangzhou, 510275, People's Republic of China.
J Hematol Oncol. 2019 Jun 7;12(1):55. doi: 10.1186/s13045-019-0748-z.
Noncoding RNAs (ncRNAs) represent a large segment of the human transcriptome and have been shown to play important roles in cellular physiology and disease pathogenesis. Increasing evidence on the functional roles of ncRNAs in cancer progression emphasizes the potential of ncRNAs for cancer treatment. Here, we summarize the roles of ncRNAs in disease relapse and resistance to current standard chemotherapy and radiotherapy; the current research progress on ncRNAs for clinical and/or potential translational applications, including the identification of ncRNAs as therapeutic targets; therapeutic approaches for ncRNA targeting; and ncRNA delivery strategies in potential clinical translation. Several ongoing clinical trials of novel RNA-based therapeutics were also emphasized. Finally, we discussed the perspectives and obstacles to different target combinations, delivery strategies, and system designs for ncRNA application. The next approved nucleic acid drug to treat cancer patients may realistically be on the horizon.
非编码 RNA（ncRNAs）是人类转录组的重要组成部分，它们在细胞生理学和疾病发病机制中发挥着重要作用。越来越多的证据表明，ncRNAs 在癌症进展中的功能作用强调了 ncRNAs 在癌症治疗中的潜力。在这里，我们总结了 ncRNAs 在疾病复发和对当前标准化疗和放疗的耐药性中的作用；目前关于 ncRNAs 的临床和/或潜在转化应用的研究进展，包括将 ncRNAs 鉴定为治疗靶点；针对 ncRNA 的治疗方法；以及 ncRNA 在潜在临床转化中的递药策略。还强调了几种正在进行的新型基于 RNA 的治疗药物的临床试验。最后，我们讨论了不同的靶点组合、递药策略和系统设计用于 ncRNA 应用的前景和障碍。下一个被批准用于治疗癌症患者的核酸药物可能即将面世。
