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肾小球肾炎治疗中精准医学的前景。

Prospects for Precision Medicine in Glomerulonephritis Treatment.

作者信息

Liu Yulu Cherry, Chun Justin

机构信息

Department of Cell Biology, Harvard Medical School, Boston, MA, USA.

Division of Nephrology, Department of Medicine, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, USA.

出版信息

Can J Kidney Health Dis. 2018 Feb 9;5:2054358117753617. doi: 10.1177/2054358117753617. eCollection 2018.

Abstract

BACKGROUND

Glomerulonephritis (GN) consists of a group of kidney diseases that are categorized based on shared histopathological features. The current classifications for GN make it difficult to distinguish the individual variability in presentation, disease progression, and response to treatment. GN is a significant cause of end-stage renal disease (ESRD), and improved therapies are desperately needed because current immunosuppressive therapies sometimes lack efficacy and can lead to significant toxicities. In recent years, the combination of high-throughput genetic approaches and technological advances has identified important regulators contributing to GN.

OBJECTIVES

In this review, we summarize recent findings in podocyte biology and advances in experimental approaches that have opened the possibility of precision medicine in GN treatment. We provide an integrative basic science and clinical overview of new developments in GN research and the discovery of potential candidates for targeted therapies in GN.

FINDINGS

Advances in podocyte biology have identified many candidates for therapeutic targets and potential biomarkers of glomerular disease. The goal of precision medicine in GN is now being pursued with recent technological improvements in genetics, accessibility of biologic and clinical information with tissue biobanks, high-throughput analysis of large-scale data sets, and new human model systems such as kidney organoids.

CONCLUSION

With advances in data collection, technologies, and experimental model systems, we now have vast tools available to pursue precision medicine in GN. We anticipate a growing number of studies integrating data from high-throughput analysis with the development of diagnostic tools and targeted therapies for GN in the near future.

摘要

背景

肾小球肾炎(GN)是一组根据共同的组织病理学特征进行分类的肾脏疾病。目前的GN分类难以区分其在临床表现、疾病进展和治疗反应方面的个体差异。GN是终末期肾病(ESRD)的重要病因,由于目前的免疫抑制疗法有时缺乏疗效且会导致严重毒性,因此迫切需要改进治疗方法。近年来,高通量基因方法与技术进步相结合,已确定了导致GN的重要调节因子。

目的

在本综述中,我们总结了足细胞生物学的最新发现以及实验方法的进展,这些进展为GN治疗中的精准医学开辟了可能性。我们提供了GN研究新进展以及GN靶向治疗潜在候选物发现的基础科学与临床综合概述。

研究结果

足细胞生物学的进展已确定了许多肾小球疾病治疗靶点和潜在生物标志物的候选物。随着遗传学技术的最新改进、生物样本库提供的生物和临床信息的可得性、大规模数据集的高通量分析以及诸如肾脏类器官等新的人类模型系统的出现,GN精准医学的目标正在逐步实现。

结论

随着数据收集、技术和实验模型系统的进步,我们现在拥有大量工具可用于GN的精准医学研究。我们预计在不久的将来,将有越来越多的研究将高通量分析数据与GN诊断工具和靶向治疗的开发相结合。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5691/5808958/fefd7de7a356/10.1177_2054358117753617-fig1.jpg

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