Al-Kindi Mohammed Nasser, Al-Khabouri Mazin Jawad, Al-Lamki Khalsa Ahmad, Palombo Flavia, Pippucci Tommaso, Romeo Giovanni, Al-Wardy Nadia Mohammed
Department of Biochemistry, College of Medicine and Health Sciences, Sultan Qaboos University, P.O. Box 35, Al-Khoud, 123, Muscat, Oman.
Department of Otolaryngology and Head and Neck Surgery, Al Nahda Hospital, Ministry of Health, Muscat, Oman.
J Genet Eng Biotechnol. 2020 Mar 2;18(1):8. doi: 10.1186/s43141-020-0021-4.
Hereditary hearing loss is a heterogeneous group of complex disorders with an overall incidence of one in every 500 newborns presented as syndromic and non-syndromic forms. Cadherin-related 23 (CDH23) is one of the listed deafness causative genes. It is found to be expressed in the stereocilia of hair cells and in the retina photoreceptor cells. Defective CDH23 have been associated mostly with prelingual severe-to-profound sensorineural hearing loss (SNHL) in either syndromic (USH1D) or non-syndromic SNHL (DFNB12) deafness. The purpose of this study was to identify causative mutations in an Omani family diagnosed with severe-profound sensorineural hearing loss by whole exome sequencing technique and analyzing the detected variant in silico for pathogenicity using several in silico mutation prediction software.
A novel homozygous missense variant, c.A7436C (p. D2479A), in exon 53 of CDH23 was detected in the family while the control samples were all negative for the detected variant. In silico mutation prediction analysis showed the novel substituted D2479A to be deleterious and protein destabilizing mutation at a conserved site on CDH23 protein.
In silico mutation prediction analysis might be used as a useful molecular diagnostic tool benefiting both genetic counseling and mutation verification. The aspartic acid 2479 alanine missense substitution might be the main disease-causing mutation that damages CDH23 function and could be used as a genetic hearing loss marker for this particular Omani family.
遗传性听力损失是一组异质性复杂疾病,在每500名新生儿中的总体发病率为1例,表现为综合征型和非综合征型。钙黏蛋白相关23(CDH23)是已列出的致聋基因之一。研究发现它在毛细胞的静纤毛和视网膜光感受器细胞中表达。有缺陷的CDH23主要与综合征型(USH1D)或非综合征型感音神经性听力损失(DFNB12)耳聋中的语前重度至极重度感音神经性听力损失(SNHL)相关。本研究的目的是通过全外显子组测序技术,在一个被诊断为重度至极重度感音神经性听力损失的阿曼家庭中鉴定致病突变,并使用几种计算机模拟突变预测软件对检测到的变异进行计算机模拟致病性分析。
在该家庭中检测到CDH23第53外显子的一个新的纯合错义变异,c.A7436C(p.D2479A),而对照样本中该检测变异均为阴性。计算机模拟突变预测分析表明,新的替代D2479A是CDH23蛋白保守位点上的有害且使蛋白质不稳定的突变。
计算机模拟突变预测分析可作为一种有用的分子诊断工具,有利于遗传咨询和突变验证。天冬氨酸2479丙氨酸错义替代可能是损害CDH23功能的主要致病突变,可作为该特定阿曼家庭遗传性听力损失的标志物。