MicroRNAs (miRNAs) are small non-coding RNAs that have been identified as key endogenous biomolecules that are able to regulate gene expression at the post-transcriptional level. The abnormal expression or function of miRNAs has been demonstrated to be closely related to the occurrence or development of various human diseases, including cancers. Regulation of these abnormal miRNAs thus holds great promise for therapeutic treatments. In this review, we summarize exogenous molecules that are able to regulate endogenous miRNAs, including small molecule regulators of miRNAs and synthetic oligonucleotides. Strategies for screening small molecule regulators of miRNAs and recently reported small molecules are introduced and summarized. Synthetic oligonucleotides including antisense miRNA oligonucleotides and miRNA mimics, as well as delivery systems for these synthetic oligonucleotides to enter cells, that regulate endogenous miRNAs are also summarized. In addition, we discuss recent applications of these small molecules and synthetic oligonucleotides in therapeutic treatments. Overall, this review aims to provide a brief synopsis of recent achievements of using both small molecule regulators and synthetic oligonucleotides to regulate endogenous miRNAs and achieve therapeutic outcomes. We envision that these regulators of endogenous miRNAs will ultimately contribute to the development of new therapies in the future.

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MicroRNA 作为调控和治疗的新型内源性靶标

MicroRNA (miRNA) 是小型非编码 RNA，已被确定为能够在转录后水平调节基因表达的关键内源生物分子。miRNA的表达或功能异常已被证明与包括癌症在内的多种人类疾病的发生或发展密切相关。因此，对这些异常 miRNA 的调节为治疗带来了巨大的希望。在这篇综述中，我们总结了能够调节内源 miRNA 的外源分子，包括 miRNA 的小分子调节剂和合成寡核苷酸。介绍并总结了筛选 miRNA 小分子调节因子和最近报道的小分子的策略。还总结了合成寡核苷酸，包括反义 miRNA 寡核苷酸和 miRNA 模拟物，以及这些合成寡核苷酸进入细胞、调节内源 miRNA 的递送系统。此外，我们还讨论了这些小分子和合成寡核苷酸在治疗中的最新应用。总的来说，这篇综述旨在简要概述使用小分子调节剂和合成寡核苷酸来调节内源性 miRNA 并实现治疗结果的最新成就。我们预计这些内源性 miRNA 的调节因子最终将有助于未来新疗法的开发。