Division of Molecular and Cellular Medicine, National Cancer Center Research Institute, Tokyo, Japan.
Department of Cellular and Molecular Biology, Graduate School of Biomedical Sciences, Hiroshima University, Hiroshima, Japan.
Cancer Sci. 2019 Apr;110(4):1140-1147. doi: 10.1111/cas.13965. Epub 2019 Feb 26.
Over the past few decades, siRNA and miRNA have attracted a great deal of attention from researchers and clinicians. These molecules have been extensively studied from the standpoint of developing biopharmaceuticals against various diseases, including heart disease, diabetes and cancers. siRNA suppresses only a single target, whereas each miRNA regulates the expression of multiple target genes. More importantly, because miRNA are also secreted from cancer cells, and their aberrant expression is associated with tumor development and progression, they represent not only therapeutic targets but also promising biomarkers for diagnosis and prognosis. Therefore, miRNA may be more effective tools against cancers, in which multiple signal pathways are dysregulated. In this review, we summarize recent progress in the development of miRNA therapeutics for the treatment of cancer patients, and describe delivery systems for oligonucleotide therapeutics.
在过去的几十年中，siRNA 和 miRNA 引起了研究人员和临床医生的极大关注。这些分子从开发针对各种疾病（包括心脏病，糖尿病和癌症）的生物制药的角度进行了广泛的研究。siRNA 仅抑制单个靶标，而每个 miRNA 都调节多个靶标基因的表达。更重要的是，由于 miRNA 也从癌细胞中分泌出来，并且它们的异常表达与肿瘤的发生和发展有关，因此它们不仅代表了治疗靶标，而且还代表了用于诊断和预后的有前途的生物标志物。因此，miRNA 可能是针对癌症的更有效工具，在癌症中，多个信号通路失调。在这篇综述中，我们总结了 miRNA 治疗剂在癌症患者治疗中的最新进展，并描述了寡核苷酸治疗剂的递药系统。
