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一个中国人家系中发现一个新型 EYA4 致病突变与 EYA4 变异的分子流行病学分析。该家系中患者患有后天性非综合征型听力损失。

Identification of a novel EYA4 likely pathogenic variant in a Chinese family with postlingual non-syndromic hearing loss and analysis of molecular epidemiology of EYA4 variants.

机构信息

Department of Otolaryngology-Head and Neck Surgery, Peking University International Hospital, 1 Shengmingyuan Road, Beijing, 100026, China.

Department of Otolaryngology-Head and Neck Surgery, First Medical Center of Chinese PLA General Hospital, Medical School of Chinese PLA, 28 Fuxing Road, Beijing, 100853, China.

出版信息

BMC Med Genomics. 2024 Oct 3;17(1):242. doi: 10.1186/s12920-024-02010-6.

Abstract

BACKGROUND

EYA4 variants are responsible for DFNA10 deafness. Due to its insidious onset and slow progression, hearing loss in autosomal dominant non-syndromic hearing loss (ADNSHL) is usually challenging to detect early in clinical settings, with limited intervention options. Genetic testing can aid in early detection of hearing loss, enabling timely intervention to reduce disability rates and improve the quality of life.

METHODS

In this study, we report the case of a Chinese family with postlingual and progressive hearing loss that was passed down for four generations. Whole-exome sequencing (WES) was performed on DNA samples from the proband. Candidate variants identified in the proband and family members were confirmed via Sanger sequencing. In silico prediction tools and co-segregation analyses were used to assess the pathogenicity of identified variants. A literature review of known EYA4 variants was performed, analysing variant frequency, distribution characteristics across different populations, and genotype-phenotype correlations.

RESULTS

We identified a novel EYA4 variant, c.1745_1748del (p.Glu582ValfsTer6), in a Chinese family with ADNSHL, and co-segregation with the family's phenotype was confirmed. The audiometry showed mid-to-high frequency downsloping hearing loss. To date, 52 pathogenic variants of EYA4 have been reported, with majority identified in Asian populations. Most observed are the missense and frameshift variants.

CONCLUSIONS

A novel variant of EYA4 was identified in a Chinese family with postlingual hearing loss, contributing to the expanding spectrum of EYA4 variants. The audiological features of EYA4 variants are highly heterogeneous and often challenging to detect early in clinical settings. Our findings highlight the significance of genetic testing in patients presenting with postlingual hearing loss.

摘要

背景

EYA4 变体是 DFNA10 耳聋的致病原因。由于其隐匿性发病和缓慢进展,常染色体显性非综合征性听力损失(ADNSHL)患者的听力损失在临床环境中很难早期发现,干预手段也很有限。基因检测有助于早期发现听力损失,从而能够及时干预,降低残疾率,提高生活质量。

方法

本研究报告了一个有四代人遗传的后天性、进行性听力损失的中国家庭。对先证者的 DNA 样本进行全外显子组测序(WES)。通过 Sanger 测序对先证者和家系成员中发现的候选变异进行确认。使用计算机预测工具和共分离分析来评估鉴定变异的致病性。对已知 EYA4 变异进行文献回顾,分析变异频率、在不同人群中的分布特征以及基因型-表型相关性。

结果

我们在一个有 ADNSHL 的中国家庭中发现了一个新的 EYA4 变异,c.1745_1748del(p.Glu582ValfsTer6),并证实与该家庭的表型共分离。听力计显示中高频下降型听力损失。迄今为止,已报道了 52 种致病性 EYA4 变异,其中大多数在亚洲人群中发现。最常见的是错义和移码变异。

结论

在一个有后天性听力损失的中国家庭中发现了 EYA4 的一个新变异,为 EYA4 变异谱的扩展做出了贡献。EYA4 变异的听力学特征高度异质性,在临床环境中通常很难早期发现。我们的发现强调了对有后天性听力损失患者进行基因检测的重要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ca6a/11447973/fb2d0307734a/12920_2024_2010_Fig1_HTML.jpg

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