Faculty of Marine Sciences, Lasbela University of Agriculture, Water and Marine Sciences (LUAWMS), Uthal, Balochistan, Pakistan.
Department of Life Sciences, School of Science, University of Management and Technology (UMT), Lahore, Pakistan.
PLoS One. 2022 Jun 16;17(6):e0268078. doi: 10.1371/journal.pone.0268078. eCollection 2022.
Hearing impairment (HI) is a heterogeneous condition that affects many individuals globally with different age groups. HI is a genetically and phenotypically heterogeneous disorder. Over the last several years, many genes/loci causing rare autosomal recessive and dominant forms of hearing impairments have been identified, involved in various aspects of ear development. In the current study, two affected individuals of a consanguineous family exhibiting autosomal recessive nonsyndromic hearing impairment (AR-NSHI) were clinically and genetically characterized. The single affected individual (IV-2) of the family was subjected to whole-exome sequencing (WES) accompanied by traditional Sanger sequencing. Clinical examinations using air conduction audiograms of both the affected individuals showed profound hearing loss across all frequencies. WES revealed a homozygous missense variant (c.44G>C) in the SIX5 gene located on chromosome 19q13.32. We report the first case of autosomal recessive NSHI due to a biallelic missense variant in the SIX5 gene. This report further supports the evidence that the SIX5 variant might cause profound HI and supports its vital role in auditory function. Identification of novel candidate genes might help in application of future gene therapy strategies that may be implemented for NSHI, such as gene replacement using cDNA, gene silencing using RNA interference, and gene editing using the CRISPR/Cas9 system.
听力障碍(HI)是一种影响全球不同年龄组许多个体的异质性疾病。HI 是一种遗传和表型异质性障碍。在过去的几年中,已经确定了许多导致罕见常染色体隐性和显性听力障碍的基因/基因座,涉及耳朵发育的各个方面。在本研究中,对一个表现出常染色体隐性非综合征性听力障碍(AR-NSHI)的近亲家族的两个受影响个体进行了临床和遗传特征分析。该家族的单一受影响个体(IV-2)接受了全外显子组测序(WES),并结合传统的 Sanger 测序。对受影响个体的空气传导听力图进行的临床检查显示所有频率均存在严重听力损失。WES 显示位于 19q13.32 染色体上的 SIX5 基因中的纯合错义变异(c.44G>C)。我们报告了首例由于 SIX5 基因的双等位基因错义变异引起的常染色体隐性 NSHI。该报告进一步支持了 SIX5 变异可能导致严重 HI 的证据,并支持其在听觉功能中的重要作用。鉴定新的候选基因可能有助于应用未来的基因治疗策略,例如使用 cDNA 进行基因替代、使用 RNA 干扰进行基因沉默以及使用 CRISPR/Cas9 系统进行基因编辑,这些策略可能用于 NSHI。