微小RNA疗法的潜力与临床研究
The Potential for microRNA Therapeutics and Clinical Research.
作者信息
Hanna Johora, Hossain Gazi S, Kocerha Jannet
机构信息
Nova Clinical Research, LLC, Bradenton, FL, United States.
Department of Chemistry and Biochemistry, Georgia Southern University, Statesboro, GA, United States.
出版信息
Front Genet. 2019 May 16;10:478. doi: 10.3389/fgene.2019.00478. eCollection 2019.
Abstract
As FDA-approved small RNA drugs start to enter clinical medicine, ongoing studies for the microRNA (miRNA) class of small RNAs expand its preclinical and clinical research applications. A growing number of reports suggest a significant utility of miRNAs as biomarkers for pathogenic conditions, modulators of drug resistance, and/or as drugs for medical intervention in almost all human health conditions. The pleiotropic nature of this class of nonprotein-coding RNAs makes them particularly attractive drug targets for diseases with a multifactorial origin and no current effective treatments. As candidate miRNAs begin to proceed toward initiation and completion of potential phase 3 and 4 trials in the future, the landscape of both diagnostic and interventional medicine will arguably continue to evolve. In this mini-review, we discuss miRNA drug discovery development and their current status in clinical trials.
摘要
随着美国食品药品监督管理局（FDA）批准的小RNA药物开始进入临床医学领域，针对微小RNA（miRNA）这类小RNA的正在进行的研究扩展了其临床前和临床研究应用。越来越多的报告表明，miRNA作为致病状况的生物标志物、耐药性调节剂和/或几乎所有人类健康状况下医学干预的药物具有显著效用。这类非蛋白质编码RNA的多效性使其成为具有多因素起源且目前尚无有效治疗方法的疾病特别有吸引力的药物靶点。随着候选miRNA在未来开始迈向潜在的3期和4期试验的启动和完成，诊断医学和介入医学的格局无疑将继续演变。在这篇小型综述中，我们讨论了miRNA药物的发现、开发及其在临床试验中的现状。
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本文引用的文献
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Expert Rev Clin Pharmacol. 2019 Feb;12(2):95-99. doi: 10.1080/17512433.2019.1567326. Epub 2019 Jan 18.
2
Niacin induces miR-502-3p expression which impairs insulin sensitivity in human adipocytes.烟酰胺诱导 miR-502-3p 的表达，从而损害人脂肪细胞的胰岛素敏感性。
Int J Obes (Lond). 2019 Jul;43(7):1485-1490. doi: 10.1038/s41366-018-0260-5. Epub 2018 Nov 27.
3
Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis.帕替拉韦，一种用于治疗遗传性转甲状腺素蛋白介导的淀粉样变性的RNA干扰疗法。
Neurodegener Dis Manag. 2019 Feb;9(1):5-23. doi: 10.2217/nmt-2018-0033. Epub 2018 Nov 27.
4
MicroRNAs as Therapeutic Agents: The Future of the Battle Against Cancer.微小 RNA 作为治疗剂：抗癌之战的未来。
Curr Top Med Chem. 2018;18(30):2544-2554. doi: 10.2174/1568026619666181120121830.
5
Current chemotherapy strategies in malignant pleural mesothelioma.恶性胸膜间皮瘤的当前化疗策略
Transl Lung Cancer Res. 2018 Oct;7(5):574-583. doi: 10.21037/tlcr.2018.04.10.
6
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CNS Neurol Disord Drug Targets. 2019;18(3):172-184. doi: 10.2174/1871527317666181114163515.
7
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8
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9
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