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肾脏疾病中的微小RNA:一种潜在的新型治疗靶点。

MicroRNAs in Renal Diseases: A Potential Novel Therapeutic Target.

作者信息

Petrillo Federica, Iervolino Anna, Zacchia Miriam, Simeoni Adelina, Masella Cristina, Capolongo Giovanna, Perna Alessandra, Capasso Giovambattista, Trepiccione Francesco

机构信息

Department of Cardiothoracic and Respiratory Science, University of Campania Luigi Vanvitelli, Naples, Italy.

Biogem S.c.a.r.l., Research Institute Gaetano Salvatore, Ariano Irpino, Italy.

出版信息

Kidney Dis (Basel). 2017 Dec;3(3):111-119. doi: 10.1159/000481730. Epub 2017 Nov 8.

Abstract

BACKGROUND

MicroRNAs (miRNAs) are a family of short noncoding RNAs that play important roles in posttranscriptional gene regulation. miRNAs inhibit target gene expression by blocking protein translation or by inducing mRNA degradation and therefore have the potential to modulate physiological and pathological processes.

SUMMARY

In the kidney, miRNAs play a role in the organogenesis and in the pathogenesis of several diseases, including renal carcinoma, diabetic nephropathy, cystogenesis, and glomerulopathies. Indeed, podocytes, but also the parietal cells of the Bowman capsule are severely affected by miRNA deregulation. In addition, several miRNAs have been found involved in the development of renal fibrosis. These experimental lines of evidence found a counterpart also in patients affected by diabetic and Ig-A nephropathies, opening the possibility of their use as biomarkers. Finally, the possibility to direct target-specific miRNA to prevent the development of renal fibrosis is encouraging potential novel therapies based on miRNA mimicking or antagonism. This review reports the main studies that investigate the role of miRNAs in the kidneys, in particular highlighting the experimental models used, their potential role as biomarkers and, finally, the most recent data on the miRNA-based therapy.

KEY MESSAGES

miRNAs are crucial regulators of cell function. They are easy to detect and represent potentially good targets for novel therapies.

摘要

背景

微小RNA(miRNA)是一类短的非编码RNA,在转录后基因调控中发挥重要作用。miRNA通过阻断蛋白质翻译或诱导mRNA降解来抑制靶基因表达,因此具有调节生理和病理过程的潜力。

总结

在肾脏中,miRNA在器官发生以及包括肾癌、糖尿病肾病、囊肿形成和肾小球病在内的多种疾病的发病机制中发挥作用。事实上,足细胞以及肾小囊壁层细胞都会受到miRNA失调的严重影响。此外,已发现几种miRNA参与肾纤维化的发展。这些实验证据在糖尿病和IgA肾病患者中也有相应发现,这为将其用作生物标志物提供了可能性。最后,将靶向特异性miRNA用于预防肾纤维化发展的可能性为基于miRNA模拟或拮抗的潜在新疗法带来了希望。本综述报告了主要研究,这些研究调查了miRNA在肾脏中的作用,特别强调了所使用的实验模型、它们作为生物标志物的潜在作用,以及最后关于基于miRNA疗法的最新数据。

关键信息

miRNA是细胞功能的关键调节因子。它们易于检测,是新型疗法的潜在良好靶点。

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Gene Therapy.基因治疗
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