Basak Indranil, Patil Ketan S, Alves Guido, Larsen Jan Petter, Møller Simon Geir
Department of Biological Sciences, St. John's University, 8000 Utopia Parkway, New York, NY, 11439, USA.
Norwegian Center for Movement Disorders, Stavanger University Hospital, 4068, Stavanger, Norway.
Cell Mol Life Sci. 2016 Feb;73(4):811-27. doi: 10.1007/s00018-015-2093-x. Epub 2015 Nov 25.
The last decade has experienced the emergence of microRNAs as a key molecular tool for the diagnosis and prognosis of human diseases. Although the focus has mostly been on cancer, neurodegenerative diseases present an exciting, yet less explored, platform for microRNA research. Several studies have highlighted the significance of microRNAs in neurogenesis and neurodegeneration, and pre-clinical studies have shown the potential of microRNAs as biomarkers. Despite this, no bona fide microRNAs have been identified as true diagnostic or prognostic biomarkers for neurodegenerative disease. This is mainly due to the lack of precisely defined patient cohorts and the variability within and between individual cohorts. However, the discovery that microRNAs exist as stable molecules at detectable levels in body fluids has opened up new avenues for microRNAs as potential biomarker candidates. Furthermore, technological developments in microRNA biology have contributed to the possible design of microRNA-mediated disease intervention strategies. The combination of these advancements, with the availability of well-defined longitudinal patient cohort, promises to not only assist in developing invaluable diagnostic tools for clinicians, but also to increase our overall understanding of the underlying heterogeneity of neurodegenerative diseases. In this review, we present a comprehensive overview of the existing knowledge of microRNAs in neurodegeneration and provide a perspective of the applicability of microRNAs as a basis for future therapeutic intervention strategies.
在过去十年中，微小RNA作为人类疾病诊断和预后的关键分子工具应运而生。尽管研究重点大多集中在癌症上，但神经退行性疾病为微小RNA研究提供了一个令人兴奋但尚未充分探索的平台。多项研究强调了微小RNA在神经发生和神经退行性变中的重要性，临床前研究也显示了微小RNA作为生物标志物的潜力。尽管如此，尚未有真正的微小RNA被确定为神经退行性疾病的真正诊断或预后生物标志物。这主要是由于缺乏精确定义的患者队列以及各个队列内部和之间的变异性。然而，微小RNA以稳定分子形式存在于体液中且可检测到这一发现，为微小RNA作为潜在生物标志物候选物开辟了新途径。此外，微小RNA生物学的技术发展有助于设计微小RNA介导的疾病干预策略。这些进展与明确的纵向患者队列的可用性相结合，有望不仅帮助临床医生开发出宝贵的诊断工具，还能增进我们对神经退行性疾病潜在异质性的全面理解。在这篇综述中，我们全面概述了微小RNA在神经退行性变方面的现有知识，并展望了微小RNA作为未来治疗干预策略基础的适用性。
