Department of ENT&HNS, "Marie Sklodowska Curie" Emergency Children's Hospital, 041434 Bucharest, Romania.
Department of Medical Genetics, Faculty of Medicine, "Titu Maiorescu" University, 031593 Bucharest, Romania.
Medicina (Kaunas). 2022 Sep 9;58(9):1252. doi: 10.3390/medicina58091252.
(1) Background: In this paper, we report on three cases of hypoacusis as part of a complex phenotype and some rare gene variants. An extensive review of literature completes the newly reported clinical and genetic information. (2) Methods: The cases range from 2- to 11-year-old boys, all with a complex clinical picture and hearing impairment. In all cases, whole exome sequencing (WES) was performed, in the first case in association with mitochondrial DNA study. (3) Results: The detected variants were: two heterozygous variants in the TWNK gene, one likely pathogenic and another of uncertain clinical significance (autosomal recessive mitochondrial DNA depletion syndrome type 7-hepatocerebral type); heterozygous variants of uncertain significance PACS2 and SYT2 genes (autosomal dominant early infantile epileptic encephalopathy) and a homozygous variant of uncertain significance in SUCLG1 gene (mitochondrial DNA depletion syndrome 9). Some of these genes have never been previously reported as associated with hearing problems. (4) Conclusions: Our cases bring new insights into some rare genetic syndromes. Although the role of TWNK gene in hearing impairment is clear and accordingly reflected in published literature as well as in the present article, for the presented gene variants, a correlation to hearing problems could not yet be established and requires more scientific data. We consider that further studies are necessary for a better understanding of the role of these variants.
(1) 背景:本文报告了三例听力下降作为复杂表型的一部分,以及一些罕见的基因变异。对文献的广泛回顾补充了新报告的临床和遗传信息。(2) 方法:这些病例的年龄从 2 岁到 11 岁不等,均具有复杂的临床表现和听力障碍。在所有情况下,均进行了全外显子组测序(WES),在第一个病例中还进行了线粒体 DNA 研究。(3) 结果:检测到的变异如下:TWNK 基因的两个杂合变异,一个可能致病,另一个临床意义不明(常染色体隐性线粒体 DNA 耗竭综合征 7-肝脑型);PACS2 和 SYT2 基因的杂合变异,意义不明(常染色体显性早发性婴儿癫痫性脑病)和 SUCLG1 基因的纯合变异,意义不明(线粒体 DNA 耗竭综合征 9)。这些基因中的一些以前从未被报道与听力问题有关。(4) 结论:我们的病例为一些罕见的遗传综合征提供了新的见解。虽然 TWNK 基因在听力下降中的作用是明确的,并相应地反映在已发表的文献以及本文中,但对于所提出的基因变异,与听力问题的相关性尚未确定,需要更多的科学数据。我们认为,需要进一步的研究来更好地理解这些变异的作用。