Surgery, School of Medicine, National University of Ireland, Galway, Ireland.
Pharm Res. 2011 Dec;28(12):3016-29. doi: 10.1007/s11095-011-0550-2. Epub 2011 Aug 5.
MiRNAs are a class of small, naturally occurring RNA molecules that play critical roles in modulating numerous biological pathways by regulating gene expression. The knowledge that miRNA expression is dysregulated in many pathological disease processes, including cancer, has led to a rapidly expanding body of literature as we try to unveil their mechanism of action. Their putative role as oncogenes or tumour suppressor genes presents a wonderful opportunity to provide targeted cancer treatment strategies. Additionally, their documented function in a host of benign diseases broadens the potential market for miRNA-based therapeutics. The present review outlines the underlying rationales for considering mi(cro)RNAs as therapeutic agents or targets. We highlight the potential of manipulating miRNAs for the treatment of many common diseases, particularly cancers. Finally, we summarize the challenges that need to be overcome to fully harness the potential of miRNA-based therapies so they become the next generation of pharmaceutical products.
miRNAs 是一类小的、天然存在的 RNA 分子，通过调节基因表达在调节众多生物途径中发挥关键作用。miRNA 表达在许多病理疾病过程中失调的知识，包括癌症，导致了大量文献的快速扩展，因为我们试图揭示它们的作用机制。它们作为癌基因或肿瘤抑制基因的假定作用为提供靶向癌症治疗策略提供了极好的机会。此外，它们在众多良性疾病中的有案可查的功能拓宽了基于 miRNA 的治疗药物的潜在市场。本综述概述了将 mi(cro)RNAs 视为治疗剂或靶标的基本原理。我们强调了操纵 miRNA 治疗许多常见疾病（特别是癌症）的潜力。最后，我们总结了需要克服的挑战，以充分利用 miRNA 治疗的潜力，使它们成为下一代药物产品。
