Laboratoire de Génétique Moléculaire, CHU Montpellier, Montpellier, France.
Service ORL, CHU Montpellier, Montpellier, France.
Sci Rep. 2017 Dec 1;7(1):16783. doi: 10.1038/s41598-017-16846-9.
Hearing loss is the most common sensory disorder and because of its high genetic heterogeneity, implementation of Massively Parallel Sequencing (MPS) in diagnostic laboratories is greatly improving the possibilities of offering optimal care to patients. We present the results of a two-year period of molecular diagnosis that included 207 French families referred for non-syndromic hearing loss. Our multi-step strategy involved (i) DFNB1 locus analysis, (ii) MPS of 74 genes, and (iii) additional approaches including Copy Number Variations, in silico analyses, minigene studies coupled when appropriate with complete gene sequencing, and a specific assay for STRC. This comprehensive screening yielded an overall diagnostic rate of 48%, equally distributed between DFNB1 (24%) and the other genes (24%). Pathogenic genotypes were identified in 19 different genes, with a high prevalence of GJB2, STRC, MYO15A, OTOF, TMC1, MYO7A and USH2A. Involvement of an Usher gene was reported in 16% of the genotyped cohort. Four de novo variants were identified. This study highlights the need to develop several molecular approaches for efficient molecular diagnosis of hearing loss, as this is crucial for genetic counselling, audiological rehabilitation and the detection of syndromic forms.
听力损失是最常见的感觉障碍,由于其高度的遗传异质性,在诊断实验室中实施大规模平行测序(MPS)极大地提高了为患者提供最佳护理的可能性。我们展示了为期两年的分子诊断结果,其中包括 207 个法国家庭,这些家庭因非综合征性听力损失而被转介。我们的多步骤策略包括(i)DFNB1 基因座分析,(ii)74 个基因的 MPS,以及(iii)包括拷贝数变异、计算机分析、适当结合完整基因测序的微基因研究在内的其他方法,以及 STRC 的特定检测。这种全面筛查的总体诊断率为 48%,在 DFNB1(24%)和其他基因(24%)之间平均分布。在 19 个不同的基因中鉴定出了致病性基因型,其中 GJB2、STRC、MYO15A、OTOF、TMC1、MYO7A 和 USH2A 的患病率较高。在基因分型队列中,16%的患者涉及到一个 Usher 基因。发现了四个新生变异。这项研究强调了需要开发几种分子方法来有效地进行听力损失的分子诊断,因为这对于遗传咨询、听力康复和综合征形式的检测至关重要。