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一种新型CLRN2变异体:扩展突变谱及其在孤立性听力障碍中的关键作用。

A novel CLRN2 variant: expanding the mutation spectrum and its critical role in isolated hearing impairment.

作者信息

Ahmad Farooq, Mahmood Arif, Almazni Ibrahim Abdullah, Shakoori Afnan Mohammed, Alhakami Fatemah, Alam Qamre, Ismail Muhammad, Umair Muhammad

机构信息

Department of Biochemistry, Faculty of Sciences, Women University Swabi Main Campus Kotha, Swabi, 23430, Khyber Pakhtunkhwa, Pakistan.

Department of Environmental, Biological and Pharmaceutical Sciences and Technologies (DiSTABiF), University of Campania 'Luigi Vanvitelli', Via Vivaldi 43, Caserta,, 81100, Italy.

出版信息

Genes Genomics. 2025 Apr;47(4):417-423. doi: 10.1007/s13258-024-01590-y. Epub 2024 Oct 24.

Abstract

BACKGROUND

Biallelic variants in the CLRN2 gene have been reported to cause autosomal recessive profound hearing impairment in humans. CLRN2 belongs to the clarin gene family that encodes a tetraspan protein that contains a cytosolic N-terminus, multiple helical transmembrane domains, and an endoplasmic reticulum membrane retention signal, TKGH, in the C-terminus. The encoded protein may be important in development and homeostasis of the inner ear and retina.

METHODS

Here, we present a consanguineous family suffering from autosomal recessive non-syndromic profound hearing impairment (HI). We employed state of the art Whole exome sequencing (WES), Sanger sequencing followed by routine bioinformatics filtration steps and homology modeling to elucidate the effect of mutation at the protein level.

RESULTS

ES followed by Sanger sequencing revealed a novel homozygous nonsense variant in the CLRN2 gene [c.414 C > A; p.Cys138*]. Furthermore, insilico protein modeling of the wildtype and mutated version of the CLRN2 protein revealed large-scale changes that predict to compromise the routine normal function of the protein.

CONCLUSION

Our finding further extends the mutations spectrum of CLRN2 gene and confirms its important role in hearing homeostasis and with developmental disorder in humans.

摘要

背景

据报道,CLRN2基因的双等位基因变异可导致人类常染色体隐性遗传性重度听力障碍。CLRN2属于clarin基因家族,该家族编码一种四跨膜蛋白,其包含一个胞质N端、多个螺旋跨膜结构域以及位于C端的内质网膜滞留信号TKGH。编码的蛋白质可能在内耳和视网膜的发育及内环境稳定中发挥重要作用。

方法

在此,我们展示了一个患有常染色体隐性非综合征性重度听力障碍(HI)的近亲家庭。我们采用了先进的全外显子组测序(WES)、桑格测序,随后进行常规生物信息学过滤步骤以及同源建模,以阐明该突变在蛋白质水平上的影响。

结果

全外显子组测序随后进行桑格测序,揭示了CLRN2基因中一个新的纯合无义变异[c.414 C>A;p.Cys138*]。此外,CLRN2蛋白野生型和突变型的计算机模拟蛋白质建模显示出大规模变化,预计这些变化会损害该蛋白质的常规正常功能。

结论

我们的发现进一步扩展了CLRN2基因的突变谱,并证实了其在人类听力内环境稳定及发育障碍中的重要作用。

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