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魏斯-克鲁什卡综合征的七种新变体及表型扩展

Seven Novel Variants of Weiss-Kruszka Syndrome and Phenotype Expansion.

作者信息

Hau Anna, Baxter Anne, Chandler Kate, Fennell Andrew, Hsieh Tzung-Chien, Krawitz Peter M, Pinner Jason, Goel Himanshu

机构信息

Hunter Genetics, Hunter New England Health Service, Newcastle, Australia.

Manchester Centre for Genomic Medicine, Manchester University NHS Foundation Trust, Saint Mary's Hospital, Manchester, UK.

出版信息

Am J Med Genet A. 2025 Feb;197(2):e63856. doi: 10.1002/ajmg.a.63856. Epub 2024 Sep 17.

Abstract

Weiss-Kruszka syndrome (WKS) is a rare genetic disorder characterized by metopic ridging, ptosis, arched eyebrows, down slanting palpebral fissures, abnormalities in the corpus callosum, cardiac malformations, and variable neurodevelopmental delay. To date, 32 individuals with a diagnosis of WKS have been reported in the literature. The syndrome is caused by a heterozygous pathogenic variant in the ZNF462 gene or a deletion of the 9p31.2 region involving ZNF462. There is significant phenotypic heterogeneity and intrafamilial variability among these patients. Our study reviewed nine patients from seven unrelated families and identified seven novel heterozygous ZNF462 variants through exome sequencing. GestaltMatcher analysis of our cohort's facial images, alongside previously published images of ZNF462 patients, demonstrated a high degree of facial similarity. Further longitudinal research is needed to delineate this rare condition's long-term health implications and adult-onset features.

摘要

魏斯-克鲁什卡综合征(WKS)是一种罕见的遗传性疾病,其特征包括额缝隆起、上睑下垂、眉弓高耸、睑裂向下倾斜、胼胝体异常、心脏畸形以及不同程度的神经发育迟缓。迄今为止,文献中已报道了32例诊断为WKS的个体。该综合征由ZNF462基因的杂合致病性变异或涉及ZNF462的9p31.2区域缺失引起。这些患者之间存在显著的表型异质性和家族内变异性。我们的研究回顾了来自七个无关家族的九名患者,并通过外显子组测序鉴定出七个新的ZNF462杂合变异。对我们队列的面部图像进行的GestaltMatcher分析,以及之前发表的ZNF462患者的图像,显示出高度的面部相似性。需要进一步的纵向研究来描述这种罕见疾病对长期健康的影响和成人发病特征。

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