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波兰一个家族中WDR35基因存在复合杂合变异的颅外胚层发育异常的产前基因诊断

Prenatal genetic diagnosis of cranioectodermal dysplasia in a Polish family with compound heterozygous variants in WDR35.

作者信息

Walczak-Sztulpa Joanna, Wawrocka Anna, Leszczynska Beata, Mikulska Boyana, Arts Heleen H, Bukowska-Olech Ewelina, Daniel Maria, Krawczynski Maciej R, Latos-Bielenska Anna, Obersztyn Ewa

机构信息

Department of Medical Genetics, Poznan University of Medical Sciences, Poznan, Poland.

Department of Pediatric Nephrology, Medical University of Warsaw, Warsaw, Poland.

出版信息

Am J Med Genet A. 2020 Oct;182(10):2417-2425. doi: 10.1002/ajmg.a.61785. Epub 2020 Aug 17.

Abstract

The ciliary chondrodysplasias represent a group of clinically and genetically heterogeneous disorders that affect skeleton development. Cilia are organelles that project from the surface of many cell types and play an important role during prenatal and postnatal human development. Cranioectodermal dysplasia (Sensenbrenner syndrome, CED) is a ciliopathy primarily characterized by craniofacial, skeletal, and ectodermal abnormalities. To date six genes have been associated with CED: IFT122, WDR35, WDR19, IFT140, IFT43, and IFT52. Prenatal diagnosis of CED is challenging, and genetic testing can facilitate making a correct diagnosis. Here, we report on a family with two male siblings affected by CED: a 3.5 year-old patient and his 2 year-old brother. Molecular analysis of the proband at 1 year of age revealed compound heterozygous variants in WDR35: c.3G>A [p.(Met1-Ala30delinsMetfsTer4)] and c.2522A>T [p.(Asp841Val)]. Ultrasound examination during the second pregnancy revealed an increased nuchal translucency of 4.5 mm and a hypoplastic nasal bone at 12 weeks of gestation. Prenatal diagnostic testing was offered because of an increased risk for chromosomal abnormalities and recurrence risk for CED. Prenatal genetic analysis of a chorionic villus sample detected the WDR35 variants previously identified in the elder brother. This is the first report of a prenatal genetic diagnosis in CED.

摘要

睫状体软骨发育异常是一组临床和遗传异质性疾病,影响骨骼发育。纤毛是从多种细胞类型表面伸出的细胞器,在人类出生前和出生后的发育过程中发挥重要作用。颅外胚层发育不良(森森布伦纳综合征,CED)是一种纤毛病,主要特征为颅面、骨骼和外胚层异常。迄今为止,已有六个基因与CED相关:IFT122、WDR35、WDR19、IFT140、IFT43和IFT52。CED的产前诊断具有挑战性,基因检测有助于做出正确诊断。在此,我们报告一个有两名受CED影响的男性同胞的家庭:一名3.5岁患者和他2岁的弟弟。对先证者1岁时的分子分析显示WDR35存在复合杂合变异:c.3G>A [p.(Met1-Ala30delinsMetfsTer4)] 和c.2522A>T [p.(Asp841Val)]。第二次怀孕期间的超声检查显示,孕12周时颈部半透明带增厚至4.5毫米,鼻骨发育不全。由于染色体异常风险增加和CED复发风险,提供了产前诊断检测。对绒毛膜绒毛样本的产前基因分析检测到了先前在哥哥身上发现的WDR35变异。这是CED产前基因诊断的首例报告。

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