Dor Yeshorim, Committee for Prevention of Jewish Genetic Diseases, Brooklyn, NY, 11211, USA.
Division of Maternal Fetal Medicine, Department of Obstetrics and Gynecology, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, 10400, Thailand.
Eur J Hum Genet. 2021 Jun;29(6):988-997. doi: 10.1038/s41431-020-00790-w. Epub 2021 Jan 4.
Nonsyndromic hearing loss is genetically heterogeneous. Despite comprehensive genetic testing, many cases remain unsolved because the clinical significance of identified variants is uncertain or because biallelic pathogenic variants are not identified for presumed autosomal recessive cases. Common synonymous variants are often disregarded. Determining the pathogenicity of synonymous variants may improve genetic diagnosis. We report a synonymous variant c.9861 C > T/p.(Gly3287=) in MYO15A in homozygosity or compound heterozygosity with another pathogenic or likely pathogenic MYO15A variant in 10 unrelated families with nonsyndromic sensorineural hearing loss. Biallelic variants in MYO15A were identified in 21 affected and were absent in 22 unaffected siblings. A mini-gene assay confirms that the synonymous variant leads to abnormal splicing. The variant is enriched in the Ashkenazi Jewish population. Individuals carrying biallelic variants involving c.9861 C > T often exhibit progressive post-lingual hearing loss distinct from the congenital profound deafness typically associated with biallelic loss-of-function MYO15A variants. This study establishes the pathogenicity of the c.9861 C > T variant in MYO15A and expands the phenotypic spectrum of MYO15A-related hearing loss. Our work also highlights the importance of multicenter collaboration and data sharing to establish the pathogenicity of a relatively common synonymous variant for improved diagnosis and management of hearing loss.
非综合征型听力损失具有遗传异质性。尽管进行了全面的基因检测,但仍有许多病例无法解释,因为已识别的变异的临床意义不确定,或者因为假定的常染色体隐性病例未识别出双等位基因致病性变异。常见的同义变异通常被忽视。确定同义变异的致病性可能会改善遗传诊断。我们报告了 10 个非综合征型感音神经性听力损失无关家族中 MYO15A 中的同义变异 c.9861C>T/p.(Gly3287=),呈纯合子或复合杂合子状态,与另一个致病性或可能致病性的 MYO15A 变异有关。在 21 名受影响的个体中鉴定出双等位基因变异,在 22 名未受影响的兄弟姐妹中未发现该变异。一个微型基因检测证实该同义变异会导致异常剪接。该变异在阿什肯纳兹犹太人群中富集。携带涉及 c.9861C>T 的双等位基因变异的个体常表现出进行性语后听力损失,与双等位基因丧失功能 MYO15A 变异通常相关的先天性重度耳聋不同。本研究确立了 c.9861C>T 变异在 MYO15A 中的致病性,并扩展了 MYO15A 相关听力损失的表型谱。我们的工作还强调了多中心合作和数据共享的重要性,以确定相对常见的同义变异的致病性,从而改善听力损失的诊断和管理。