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全外显子组测序在肾脏疾病基因诊断中的应用及其临床价值。

Genetic diagnosis of kidney disease by whole exome sequencing and its clinical application.

机构信息

Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Republic of Korea.

3billion, Inc., Seoul, Republic of Korea.

出版信息

Clin Genet. 2023 Sep;104(3):298-312. doi: 10.1111/cge.14382. Epub 2023 Jun 4.

Abstract

The genetic spectrum of genetic kidney diseases (GKD) and the application of genetic diagnoses to patient care were assessed by whole exome sequencing (WES) of the DNA of 172 pediatric or adult patients with various kidney diseases. WES diagnosed genetic diseases in 63 (36.6%) patients. The diagnostic yields in patients with glomerulopathy were 33.8% (25/74 pts) due to variants in 10 genes, 58.8% (20/34) in patients with tubulointerstitial disease due to variants in 18 genes, 33.3% (15/45) in patients with cystic disease/ciliopathy due to variants in 10 genes, 18.2% (2/11) in patients with congenital anomalies of the kidneys and urinary tract (CAKUT) due to variants in two genes, and 12.5% (1/8) in patients with end stage kidney disease (ESKD). The diagnosis rate was high in patients aged <1-6 years (46-50.0%), and low in patients aged ≥40 years (9.1%). Renal phenotype was reclassified in 10 (15.9%) of 63 patients and clinical management altered in 10 (15.9%) of 63 patients after genetic diagnosis. In conclusion, these findings demonstrated the diagnostic utility of WES and its effective clinical application in patients, with various kinds of kidney diseases, across the different age groups.

摘要

通过对 172 名患有各种肾脏疾病的儿科或成年患者的 DNA 进行全外显子组测序 (WES),评估了遗传肾脏疾病 (GKD) 的遗传谱以及遗传诊断在患者治疗中的应用。WES 诊断出 63 名(36.6%)患者存在遗传疾病。由于 10 个基因中的变异,肾小球疾病患者的诊断率为 33.8%(25/74 例);由于 18 个基因中的变异,肾小管间质性疾病患者的诊断率为 58.8%(20/34 例);由于 10 个基因中的变异,囊性疾病/纤毛病患者的诊断率为 33.3%(15/45 例);由于 2 个基因中的变异,先天性肾和泌尿道异常 (CAKUT) 患者的诊断率为 18.2%(2/11 例);由于 2 个基因中的变异,终末期肾病 (ESKD) 患者的诊断率为 12.5%(1/8 例)。年龄 <1-6 岁的患者诊断率较高(46-50.0%),年龄≥40 岁的患者诊断率较低(9.1%)。在 63 名患者中,有 10 名(15.9%)患者的肾脏表型得到重新分类,在 63 名患者中有 10 名(15.9%)患者的临床管理发生了改变。总之,这些发现证明了 WES 的诊断效用及其在不同年龄段、各种肾脏疾病患者中的有效临床应用。

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