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YY1 突变导致 Gabriele-de Vries 综合征的临床分析及文献复习。

Clinical analysis of Gabriele-de Vries caused by YY1 mutations and literature review.

机构信息

Department of Pediatric Rehabilitation Medicine, Children's Hospital Affiliated to Zhengzhou University, Zhengzhou, China.

Henan Neurodevelopment Engineering Research Center for Children, Henan Key Laboratory of Children's Genetics and Metabolic Diseases, Children's Hospital Affiliated to Zhengzhou University, Zhengzhou, China.

出版信息

Mol Genet Genomic Med. 2024 Jan;12(1):e2281. doi: 10.1002/mgg3.2281. Epub 2023 Sep 1.

Abstract

BACKGROUND

Gabriele-de Vries syndrome is a rare autosomal dominant genetic disease characterized by global development delay/intellectual disability, delayed language development, feeding difficulties, and distinctive facial dysmorphism. It is caused by pathogenic variants in YY1.

METHODS

The current report describes a female patient with motor delay and a facial dysmorphism phenotype. We identified pathogenic mutations in the patient by whole-exome sequencing and confirmed them by Sanger sequencing.

RESULTS

A novel heterozygous frameshift mutation NM_003403.5:c.458_476del (p. V153fs*97) in the YY1 gene was detected in the proband. Finally, we provide a case-based review of the clinical features associated with Gabriele-de Vries syndrome. A total of 28 patients with genetic abnormalities and clinical phenotypes have been reported in the literature thus far.

CONCLUSIONS

The mutation site is reported for the first time, and its discovery would expand the mutation spectrum of the YY1 gene. The main clinical manifestations of Gabriele-de Vries syndrome are developmental delay/intellectual disability, craniofacial dysplasia, intrauterine growth delay, low birth weight, feeding difficulties, and rare congenital malformations. Genetic tests are crucial techniques for its diagnosis because of its nonspecific clinical manifestations.

摘要

背景

Gabriele-de Vries 综合征是一种罕见的常染色体显性遗传疾病,其特征为全面发育迟缓/智力障碍、语言发育迟缓、喂养困难和独特的面部畸形。它由 YY1 中的致病性变异引起。

方法

本报告描述了一名存在运动迟缓及面部畸形表型的女性患者。我们通过全外显子组测序在患者中鉴定出致病性突变,并通过 Sanger 测序进行了确认。

结果

在该先证者中检测到 YY1 基因中的新型杂合移码突变 NM_003403.5:c.458_476del(p. V153fs*97)。最后,我们基于该病例对与 Gabriele-de Vries 综合征相关的临床特征进行了综述。迄今为止,文献中已有 28 例具有遗传异常和临床表型的患者被报道。

结论

该突变位点为首次报道,其发现扩展了 YY1 基因的突变谱。Gabriele-de Vries 综合征的主要临床表现为发育迟缓/智力障碍、颅面畸形、宫内生长迟缓、低出生体重、喂养困难和罕见的先天性畸形。由于其临床表现不具特异性,因此遗传检测是其诊断的关键技术。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ca22/10767417/4144634f94cf/MGG3-12-e2281-g004.jpg

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