一名男性新生儿纤毛症表型中的 WDR44 功能缺失启动子缺失。

IF 1.7 4区 生物学 Q3 GENETICS & HEREDITY American Journal of Medical Genetics Part A Pub Date : 2024-09-05 DOI:10.1002/ajmg.a.63861
Tam P Sneddon, Kelly L Gilmore, Mai Xiong, Karen E Weck, Bradford C Powell, Neeta L Vora
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引用次数: 0

摘要

最近,WDR44 基因的功能增益变异与 X 连锁纤毛症相关的神经发育表型有关。在此,我们报告了在基因组测序产前基因诊断(PrenatalSEQ)多中心研究中一名男性胎儿的基因组序列中发现的 WDR44 功能缺失(LOF)变体。其表型与所描述的 X 连锁纤毛虫病一致,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、颅面畸形和渗出。这是首例在产前表现的受影响个体中发现WDR44 LOF变体的报告,并支持LOF是X连锁WDR44纤毛症相关表型的一种机制。
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WDR44 Loss-of-Function Promoter Deletion in a Male Newborn With a Ciliopathy Phenotype.

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.

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来源期刊
CiteScore
3.50
自引率
5.00%
发文量
432
审稿时长
2-4 weeks
期刊介绍: The American Journal of Medical Genetics - Part A (AJMG) gives you continuous coverage of all biological and medical aspects of genetic disorders and birth defects, as well as in-depth documentation of phenotype analysis within the current context of genotype/phenotype correlations. In addition to Part A , AJMG also publishes two other parts: Part B: Neuropsychiatric Genetics , covering experimental and clinical investigations of the genetic mechanisms underlying neurologic and psychiatric disorders. Part C: Seminars in Medical Genetics , guest-edited collections of thematic reviews of topical interest to the readership of AJMG .
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