Department of Biomedical Sciences, University of Ulsan College of Medicine, Asan Medical Center, Seoul 05505, Korea.
Int J Mol Sci. 2017 Dec 27;19(1):65. doi: 10.3390/ijms19010065.
MicroRNAs (miRs, miRNAs) are regulatory small noncoding RNAs, with their roles already confirmed to be important for post-transcriptional regulation of gene expression affecting cell physiology and disease development. Upregulation of a cancer-causing miRNA, known as oncogenic miRNA, has been found in many types of cancers and, therefore, represents a potential new class of targets for therapeutic inhibition. Several strategies have been developed in recent years to inhibit oncogenic miRNAs. Among them is a direct approach that targets mature oncogenic miRNA with an antisense sequence known as antimiR, which could be an oligonucleotide or miRNA sponge. In contrast, an indirect approach is to block the biogenesis of miRNA by genome editing using the CRISPR/Cas9 system or a small molecule inhibitor. The development of these inhibitors is straightforward but involves significant scientific and therapeutic challenges that need to be resolved. In this review, we summarize recent relevant studies on the development of miRNA inhibitors against cancer.
微小 RNA（miRs，miRNAs）是调节性小非编码 RNA，其作用已被证实对于影响细胞生理学和疾病发展的基因表达的转录后调控非常重要。在许多类型的癌症中发现了致癌 miRNA 的上调，因此代表了治疗抑制的新潜在靶标。近年来已经开发了几种抑制致癌 miRNA 的策略。其中一种是直接方法，该方法使用称为 antimiR 的反义序列靶向成熟的致癌 miRNA，该反义序列可以是寡核苷酸或 miRNA 海绵。相比之下，间接方法是通过使用 CRISPR/Cas9 系统或小分子抑制剂进行基因组编辑来阻断 miRNA 的生物发生。这些抑制剂的开发很直接，但涉及需要解决的重大科学和治疗挑战。在这篇综述中，我们总结了最近关于针对癌症的 miRNA 抑制剂开发的相关研究。
