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一名患者携带 GPC3 基因的独特移码突变,导致辛普森-戈尔比-比姆综合征,表现为颅缝早闭、阴茎阴囊型尿道下裂和巨大前列腺囊。

A patient with a unique frameshift mutation in GPC3, causing Simpson-Golabi-Behmel syndrome, presenting with craniosynostosis, penoscrotal hypospadias, and a large prostatic utricle.

机构信息

Department of Cellular and Structural Biology, School of Medicine, The University of Texas Health Science Center at San Antonio, San Antonio, Texas.

出版信息

Am J Med Genet A. 2013 Dec;161A(12):3121-5. doi: 10.1002/ajmg.a.36086. Epub 2013 Oct 2.

DOI:10.1002/ajmg.a.36086
PMID:24115482
Abstract

We present a Hispanic male with the clinical and molecular diagnosis of Simpson-Golabi-Behmel syndrome (SGBS). The patient was born with multiple anomalies not entirely typical of SGBS patients, including penoscrotal hypospadias, a large prostatic utricle, and left coronal craniosynostosis. In addition, he demonstrated endocrine anomalies including a low random cortisol level suspicious for adrenal insufficiency and low testosterone level. To our knowledge, this is the first report of a prostatic utricle in SGBS and the second report of craniosynostosis. The unique disease-causing mutation likely arose de novo in the mother. It is a deletion-insertion that leads to a frameshift at the p.p. S359 [corrected] residue of GPC3 and a premature stop codon after five more amino acids. p. S359 [corrected] is the same residue that is normally cleaved by the Furin convertase, although the significance of this novel mutation with respect to the patient's multiple anomalies is unknown. We present this case as the perinatal course of a patient with unique features of SGBS and a confirmed molecular diagnosis.

摘要

我们呈现了一位具有辛普森-戈拉比-贝姆尔综合征(SGBS)临床和分子诊断的西班牙裔男性患者。该患者出生时就存在多种非典型的 SGBS 患者的畸形,包括阴茎阴囊型尿道下裂、巨大的前列腺囊和左侧冠状缝早闭。此外,他还表现出内分泌异常,包括随机皮质醇水平低,疑似肾上腺功能不全和睾酮水平低。据我们所知,这是首例 SGBS 患者前列腺囊的报告,也是第二例颅缝早闭的报告。该独特的致病突变很可能是在母亲体内新发生的。它是一个移码突变,导致 GPC3 的 p.p. S359[校正]残基发生移码,并在接下来的五个氨基酸后产生一个过早的终止密码子。p.S359[校正]是正常情况下被 Furin 转化酶切割的残基,尽管这个新突变与患者的多种畸形之间的关系尚不清楚。我们呈现这个病例是因为该患者具有 SGBS 的独特特征和明确的分子诊断,同时具有围产期病程。

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