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一名患有纤毛病表型的男性新生儿中WDR44功能缺失性启动子缺失

WDR44 Loss-of-Function Promoter Deletion in a Male Newborn With a Ciliopathy Phenotype.

作者信息

Sneddon Tam P, Gilmore Kelly L, Xiong Mai, Weck Karen E, Powell Bradford C, Vora Neeta L

机构信息

Department of Pathology and Laboratory Medicine, University of North Carolina, Chapel Hill, North Carolina, USA.

Department of Genetics, University of North Carolina, Chapel Hill, North Carolina, USA.

出版信息

Am J Med Genet A. 2025 Jan;197(1):e63861. doi: 10.1002/ajmg.a.63861. Epub 2024 Sep 5.

Abstract

Gain-of-function variants in the WDR44 gene have recently been associated with an X-linked ciliopathy-related neurodevelopmental phenotype. Here, we report on a WDR44 loss-of-function (LOF) variant identified in the genome sequence from a male fetus enrolled in the Prenatal Genetic Diagnosis by Genomic Sequencing (PrenatalSEQ) multicenter study. The phenotype is consistent with the described X-linked ciliopathy that includes developmental delay, microcephaly, congenital heart defects, kidney abnormalities, cryptorchidism, musculoskeletal abnormalities, craniofacial dysmorphism, and effusions. This is the first report of a WDR44 LOF variant in an affected individual with a prenatal presentation and supports LOF as a mechanism for the X-linked WDR44 ciliopathy-related phenotype.

摘要

WDR44基因的功能获得性变异最近被发现与一种X连锁的与纤毛病相关的神经发育表型有关。在此,我们报告了在参与基因组测序产前基因诊断(PrenatalSEQ)多中心研究的一名男性胎儿的基因组序列中鉴定出的一个WDR44功能丧失(LOF)变异。该表型与所描述的X连锁纤毛病一致,包括发育迟缓、小头畸形、先天性心脏缺陷、肾脏异常、隐睾、肌肉骨骼异常、颅面畸形和积液。这是关于一名产前表现的受影响个体中WDR44 LOF变异的首次报告,并支持LOF作为X连锁WDR44纤毛病相关表型的一种机制。

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