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ZNF526基因的新型复合杂合变异导致丹蒂奇-诺韦利神经发育综合征:一例报告及文献综述

Novel Compound Heterozygous Variants in ZNF526 Causing Dentici-Novelli Neurodevelopmental Syndrome: A Case Report and Literature Review.

作者信息

Li Shaoxin, Fang Hui, Li Hong, Peng Min, Bao Jinsong, Cai Yunfei, Chen Jing, Li Zhige

机构信息

Department of Rehabilitation, Anhui Provincial Children's Hospital, Hefei, China.

Chigene (Beijing) Translational Medical Research Center Co., Beijing, China.

出版信息

Mol Genet Genomic Med. 2025 Apr;13(4):e70089. doi: 10.1002/mgg3.70089.

Abstract

BACKGROUND

The ZNF526 gene encodes a ubiquitously expressed Kruppel-type zinc finger protein crucial in transcriptional regulation. Recent studies suggest that biallelic pathogenic variants in ZNF526 may lead to Dentici-Novelli neurodevelopmental syndrome, characterized by microcephaly, developmental delay, epilepsy, and ocular anomalies. To date, phenotypic details have been reported for only six patients with ZNF526 variants.

METHODS

This study gathered clinical information and genetic data from a child with neurodevelopmental disorders. A three-dimensional protein model was employed to predict variant effects on protein structure. A literature review was conducted to compare this case with previously reported cases, analyzing clinical features and genetic findings.

RESULTS

The proband, a 7-month-old girl, exhibited developmental delay, microcephaly, limb hypotonia, abnormal brain imaging, and seizures. Chromosomal karyotype analysis and copy number variation analyses were normal. Whole exome sequencing revealed two heterozygous variants in the ZNF526 gene (NM_133444.3): c.1426del (p.Val476Phefs*9), a de novo frameshift variant, and c.1513T;> C (p.Cys505Arg), inherited from her mother. These previously unreported variants are on separate alleles, forming a compound heterozygous state correlated with the clinical presentation. Ocular anomalies were absent, while café-au-lait spots may represent a novel feature. Among 12 cases of Dentici-Novelli neurodevelopmental syndrome, 11 unique ZNF526 variants have been identified, with loss-of-function variants possibly linked to seizures.

CONCLUSION

This study describes the youngest patient with Dentici-Novelli neurodevelopmental syndrome, broadening the ZNF526 mutation spectrum and detailing the associated clinical profile. These findings are valuable for genetic diagnosis and family counseling in cases of this syndrome.

摘要

背景

ZNF526基因编码一种在转录调控中起关键作用的普遍表达的克鲁ppel型锌指蛋白。最近的研究表明,ZNF526基因的双等位基因致病性变异可能导致登蒂奇-诺韦利神经发育综合征,其特征为小头畸形、发育迟缓、癫痫和眼部异常。迄今为止,仅报道了6例携带ZNF526变异的患者的表型细节。

方法

本研究收集了一名神经发育障碍儿童的临床信息和基因数据。采用三维蛋白质模型预测变异对蛋白质结构的影响。进行文献综述以将该病例与先前报道的病例进行比较,分析临床特征和基因发现。

结果

先证者为一名7个月大的女孩,表现出发育迟缓、小头畸形、肢体肌张力减退、脑部影像学异常和癫痫发作。染色体核型分析和拷贝数变异分析均正常。全外显子组测序在ZNF526基因(NM_133444.3)中发现了两个杂合变异:c.1426del(p.Val476Phefs*9),一个新生移码变异,以及c.1513T;>C(p.Cys505Arg),从她母亲那里遗传而来。这些先前未报道的变异位于不同的等位基因上,形成了与临床表现相关的复合杂合状态。未发现眼部异常,而咖啡斑可能是一个新特征。在12例登蒂奇-诺韦利神经发育综合征病例中,已鉴定出11种独特的ZNF526变异,功能丧失变异可能与癫痫发作有关。

结论

本研究描述了最年轻的登蒂奇-诺韦利神经发育综合征患者,拓宽了ZNF526突变谱并详细阐述了相关的临床特征。这些发现对于该综合征病例的基因诊断和遗传咨询具有重要价值。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/63b7/11976872/955634d72e7e/MGG3-13-e70089-g003.jpg

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