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遗传修饰物在肾小管间质性肾脏疾病中的作用。

A Role for Genetic Modifiers in Tubulointerstitial Kidney Diseases.

机构信息

Human Genetics & Genomic Medicine, University of Southampton, Southampton SO16 6YD, UK.

Wessex Kidney Centre, Queen Alexandra Hospital, Portsmouth Hospitals NHS Trust, Portsmouth PO6 3LY, UK.

出版信息

Genes (Basel). 2023 Aug 3;14(8):1582. doi: 10.3390/genes14081582.

Abstract

With the increased availability of genomic sequencing technologies, the molecular bases for kidney diseases such as nephronophthisis and mitochondrially inherited and autosomal-dominant tubulointerstitial kidney diseases (ADTKD) has become increasingly apparent. These tubulointerstitial kidney diseases (TKD) are monogenic diseases of the tubulointerstitium and result in interstitial fibrosis and tubular atrophy (IF/TA). However, monogenic inheritance alone does not adequately explain the highly variable onset of kidney failure and extra-renal manifestations. Phenotypes vary considerably between individuals harbouring the same pathogenic variant in the same putative monogenic gene, even within families sharing common environmental factors. While the extreme end of the disease spectrum may have dramatic syndromic manifestations typically diagnosed in childhood, many patients present a more subtle phenotype with little to differentiate them from many other common forms of non-proteinuric chronic kidney disease (CKD). This review summarises the expanding repertoire of genes underpinning TKD and their known phenotypic manifestations. Furthermore, we collate the growing evidence for a role of modifier genes and discuss the extent to which these data bridge the historical gap between apparently rare monogenic TKD and polygenic non-proteinuric CKD (excluding polycystic kidney disease).

摘要

随着基因组测序技术的日益普及,肾单位肾疾病(如肾单位纤毛病和线粒体遗传及常染色体显性遗传性肾小管间质性肾病(ADTKD))等疾病的分子基础变得越来越明显。这些肾小管间质性肾病(TKD)是肾小管间质性的单基因疾病,导致间质纤维化和肾小管萎缩(IF/TA)。然而,单基因遗传本身并不能充分解释肾衰竭和肾外表现的高度可变发作。即使在具有共同环境因素的家族中,携带相同致病性变异的同一假定单基因中的个体之间的表型也有很大差异。虽然疾病谱的极端可能具有典型在儿童期诊断的戏剧性综合征表现,但许多患者表现出更微妙的表型,与许多其他常见形式的非蛋白尿性慢性肾病(CKD)几乎没有区别。这篇综述总结了 TKD 相关基因及其已知表型表现的不断扩大的基因库。此外,我们整理了越来越多的关于修饰基因作用的证据,并讨论了这些数据在多大程度上弥合了明显罕见的单基因 TKD 和非蛋白尿性多基因 CKD(不包括多囊肾病)之间的历史差距。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c6b1/10454709/0fe11338181f/genes-14-01582-g004.jpg

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