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全外显子组测序揭示了 OSGEP 基因中的一个新型纯合错义变异:伊朗一例 Galloway-Mowat 综合征。

Whole-exome sequencing revealed a novel homozygous missense variant in OSGEP gene: a case report of Galloway-Mowat syndrome in Iran.

机构信息

Fetal Health Research Center, Hope Generation Foundation, Tehran, Iran.

Personalized Medicine and Genometabolomics Research Center, Hope Generation Foundation, Tehran, Iran.

出版信息

CEN Case Rep. 2023 Nov;12(4):374-377. doi: 10.1007/s13730-023-00775-w. Epub 2023 Mar 1.

Abstract

Galloway-Mowat syndrome is a rare autosomal-recessive genetic disorder that is characterized by variety of complications such as neurological abnormalities and early-onset progressive kidney disease. Studies have been shown that pathogenic mutations in genes that belong to the KEOPS complex lead to Galloway-Mowat syndrome. Several pathogenic mutations in OSGEP gene, a member of the KEOPS complex, have been detected in Galloway-Mowat syndrome. Here we describe a 12-year-old male with intellectual disability, poor speech, seizures, microcephaly, and nephrotic syndrome that were in favor of Galloway-Mowat syndrome, born to a healthy Iranian consanguineous parents. Extracted genomic DNA from blood sample was used to perform whole-exome sequencing in the patient. The mutational screening revealed a novel homozygote OSGEP gene missense variant. Our finding established whole-exome sequencing as a valuable technic for the detection of rare variants.

摘要

高威-莫瓦特综合征是一种罕见的常染色体隐性遗传疾病,其特征是多种并发症,如神经发育异常和早发性进行性肾病。研究表明,属于 KEOPS 复合物的基因突变会导致高威-莫瓦特综合征。在高威-莫瓦特综合征患者中已经检测到 KEOPS 复合物的一个成员 OSGEP 基因的几个致病性突变。在这里,我们描述了一名 12 岁男性,他患有智力残疾、言语障碍、癫痫、小头畸形和肾病综合征,这些都有利于高威-莫瓦特综合征,他出生于一对健康的伊朗近亲父母。从血样中提取基因组 DNA,用于对患者进行全外显子组测序。突变筛查显示了一种新型的 OSGEP 基因纯合错义变异。我们的发现确立了全外显子组测序作为一种检测罕见变异的有价值的技术。

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