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沙特男孩的 tectonic(TCTN1)基因相关 Joubert 综合征的临床和分子特征。

Clinical and molecular characteristics of tectonic (TCTN1) gene-related Joubert syndrome in a Saudi boy.

机构信息

Department of Neuroscience, King Faisal Specialist Hospital & Research Centre, Jeddah, Saudi Arabia.

College of Medicine, King Saud bin Abdulaziz University for Health Sciences, Jeddah, Saudi Arabia; King Abdullah International Medical Research Center, Jeddah, Saudi Arabia; Pediatric Department, Ministry of National Guard Health Affairs, Jeddah, Saudi Arabia.

出版信息

Brain Dev. 2022 Apr;44(4):299-302. doi: 10.1016/j.braindev.2021.12.005. Epub 2021 Dec 31.

Abstract

Joubert syndrome (OMIM:609863) is a hereditary disorder characterized by hypotonia, developmental delay, and a distinctive cerebellar and brain stem malformation known as the molar tooth sign. Variants in tectonic genes TCTN1-3 have been described in a few patients with Joubert syndrome. Furthermore, Joubert syndrome attributed to variants in the TCTN1 (NM_001082538.2) gene has been only described in two reports. This report expands the clinical variability and molecular characterization of an emerging novel causative gene for Joubert syndrome in a Saudi boy born to non-consanguineous marriage with a c.1418del p.(Pro473Leufs*42) and c.800A > G p.(Tyr267Cys) representing a novel compound heterozygous variant of the TCTN1 gene identified by whole-exome sequencing and confirmed by Sanger sequencing. This is the first report of compound heterozygous Joubert syndrome type 13 from Saudi Arabia.

摘要

杰伯综合征(OMIM:609863)是一种遗传性疾病,其特征为张力减退、发育迟缓以及一种特征性的小脑和脑干畸形,称为磨牙齿状标志。在少数杰伯综合征患者中已描述了 tectonic 基因 TCTN1-3 的变体。此外,归因于 TCTN1(NM_001082538.2)基因突变的杰伯综合征仅在两份报告中描述过。本报告扩展了一名沙特男孩的临床变异性和分子特征,该男孩出生于非近亲结婚,携带 c.1418del p.(Pro473Leufs*42)和 c.800A>G p.(Tyr267Cys),这是一种新型的 TCTN1 基因突变的复合杂合变体,通过外显子组测序鉴定,并通过 Sanger 测序确认。这是来自沙特阿拉伯的第 13 种复合杂合性杰伯综合征的首例报告。

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