Nanjing University Medical School, Nanjing Drum Tower Hospital, Department of Obstetrics and Gynecology , Nanjing 210008, Jiangsu , China +86 25 8330 4616 ; +86 25 8330 4616 ;
Expert Opin Investig Drugs. 2014 Mar;23(3):347-55. doi: 10.1517/13543784.2014.867327. Epub 2013 Dec 20.
microRNA (miRNA) regulates target gene expression to influence many physiological and pathophysiological processes. The liver-specific miRNA, miR-122, contributes to liver function and plays a very important role in hepatic diseases including the viral hepatitis C (HCV). For this reason, developing an miR-122-related clinical application could be very useful in managing or treating many hepatic disorders.
This review introduces the basic concepts of miRNA and miR-122. It also discusses the possibility of miR-122 as a biomarker and summarizes the results of anti-miR-122 treatment from basic research to a Phase IIa clinical trial. Furthermore, the authors discuss the potential opportunities and challenges found in clinical trials with miravirsen.
miR-122 may be a useful biomarker as both a diagnostic and prognostic tool. Furthermore, miravirsen is a novel treatment with great potential for hepatic disease treatment, especially in HCV. However, there is certainly the need for future investigations to better determine whether miR-122 is really specific for liver. It is also important to elucidate whether miR-122 is actually specific for HCV genome and further investigate the therapeutic potential of miravirsen. Only once these studies have been completed can anti-miR-122 treatment potentially enter the clinical practice.
microRNA（miRNA）通过调节靶基因的表达来影响许多生理和病理生理过程。肝脏特异性 miRNA，miR-122，有助于肝脏功能，在包括丙型肝炎病毒（HCV）在内的多种肝脏疾病中发挥着非常重要的作用。因此，开发与 miR-122 相关的临床应用可能在管理或治疗许多肝脏疾病方面非常有用。
本文介绍了 miRNA 和 miR-122 的基本概念。它还讨论了 miR-122 作为生物标志物的可能性，并总结了从基础研究到 IIa 期临床试验的抗 miR-122 治疗的结果。此外，作者还讨论了 miravirsen 临床试验中发现的潜在机遇和挑战。
miR-122 可能是一种有用的生物标志物，既是诊断标志物，也是预后标志物。此外，miravirsen 是一种新型治疗药物，对肝脏疾病的治疗具有巨大潜力，特别是对 HCV。然而，未来肯定需要进一步研究，以更好地确定 miR-122 是否真的对肝脏具有特异性。阐明 miR-122 是否真的对 HCV 基因组具有特异性，并进一步研究 miravirsen 的治疗潜力也很重要。只有在这些研究完成后，抗 miR-122 治疗才有可能进入临床实践。
