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伴有颅缝早闭的CDC45相关迈耶-戈林综合征的进一步描述及文献综述。

Further delineation of CDC45-related Meier-Gorlin syndrome with craniosynostosis and review of literature.

作者信息

Ting Chun Yi, Bhatia Neha Singh, Lim Jiin Ying, Goh Chew-Yin Jasmine, Vasanwala Rashida Farhad, Ong Caroline Choo-Phaik, Seow Wan Tew, Yeow Vincent Kok-Leng, Ting Teck Wah, Ng Ivy Swee-Lian, Jamuar Saumya Shekhar

机构信息

Department of Paediatrics, KK Women's and Children's Hospital, Singapore.

Department of Paediatrics, KK Women's and Children's Hospital, Singapore; Division of Genetics, Children's Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY, USA.

出版信息

Eur J Med Genet. 2020 Feb;63(2):103652. doi: 10.1016/j.ejmg.2019.04.009. Epub 2019 Apr 13.

Abstract

Meier-Gorlin syndrome (MGS) is a rare autosomal recessive disorder characterized by the triad of short stature, microtia and absent or small patellae. We report on a patient with MGS secondary to biallelic mutations in CDC45 detected on whole exome sequencing (WES). Patients with MGS caused by mutations in CDC45 display a distinct phenotype characterized by craniosynostosis and anorectal malformation. Our patient had craniosynostosis, anorectal malformation and short stature, but did not have the microtia or patella hypoplasia. Our report also highlights the value of WES in aiding diagnosis of patients with rare genetic diseases. In conclusion, our case report and review of the literature illustrates the unique features of CDC45-related MGS as well as the benefits of WES in reducing the diagnostic odyssey for patients with rare genetic disorders.

摘要

迈尔-戈林综合征(MGS)是一种罕见的常染色体隐性疾病,其特征为身材矮小、小耳畸形以及髌骨缺如或发育不全三联征。我们报告了一名通过全外显子组测序(WES)检测到双等位基因CDC45突变继发MGS的患者。由CDC45突变引起的MGS患者表现出以颅缝早闭和肛门直肠畸形为特征的独特表型。我们的患者有颅缝早闭、肛门直肠畸形和身材矮小,但没有小耳畸形或髌骨发育不全。我们的报告还强调了WES在辅助诊断罕见遗传病患者方面的价值。总之,我们的病例报告及文献复习阐述了CDC45相关MGS的独特特征以及WES在减少罕见遗传病患者诊断历程方面的益处。

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