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利用肾脏类器官深入了解遗传肾脏疾病。

Advances in our understanding of genetic kidney disease using kidney organoids.

机构信息

Murdoch Children's Research Institute, Flemington Rd., Parkville, VIC, Australia.

Department of Anatomy and Neuroscience, The University of Melbourne, Melbourne, VIC, Australia.

出版信息

Pediatr Nephrol. 2020 Jun;35(6):915-926. doi: 10.1007/s00467-019-04259-x. Epub 2019 May 7.

Abstract

A significant proportion of kidney disease presenting in childhood is likely genetic in origin with a growing number of genes implicated in its development. However, many children may have changes in previously undescribed or unrecognised genes. The recent development of methods for generating human kidney organoids from human pluripotent stem cells has the potential to substantially change the rate of diagnosis and the development of new treatments for some forms of genetic kidney disease. In this review, we discuss how accurately a kidney organoid models the human kidney, identifying the strengths and weaknesses of these potentially patient-derived models of renal disease.

摘要

相当一部分儿童期出现的肾脏疾病可能具有遗传基础,越来越多的基因被牵连到其发病机制中。然而,许多儿童可能存在以前未描述或未识别的基因变化。最近,从人类多能干细胞生成人类肾脏类器官的方法的发展,有可能极大地改变某些遗传性肾脏疾病的诊断率和新疗法的研发速度。在这篇综述中,我们讨论了肾脏类器官在多大程度上模拟了人类肾脏,并确定了这些潜在的源自患者的肾脏疾病模型的优缺点。

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