Petropoulou Evmorfia, Soltani Mohammadhossein, Firoozabadi Ali Dehghani, Namayandeh Seyedeh Mahdieh, Crockford Jade, Maroofian Reza, Jamshidi Yalda
Cardiovascular and Cell Sciences Institute, St George's University of London, Cranmer Terrace, London, UK.
Yazd Cardiovascular Research Center, Shahid Sadoughi University of Medical Science, Yazd, Iran.
Eur J Med Genet. 2017 Sep;60(9):485-488. doi: 10.1016/j.ejmg.2017.06.008. Epub 2017 Jun 19.
Familial dilated cardiomyopathy (DCM) is characterized by ventricular dilation and depressed myocardial performance. It is a genetically heterogeneous disorder associated with mutations in over 60 genes. We carried out whole exome sequencing in combination with cardiomyopathy-related gene-filtering on two affected family members to identify the possible causative mutation in a consanguineous Iranian family with DCM. Two novel variants in cardiomyopathy-related genes were identified: c.247 A > C; p.N83H in the Troponin T Type 2 gene (TNNT2) and c.2863G > A; p.D955N in the Myosin Heavy Polypeptide 7 gene (MYH7). Sanger sequencing and co-segregation analysis in the remaining family members supported the coexistence of these digenic mutations in affected members of the family. Carriers of either variant alone were asymptomatic. In summary, we find that digenic inheritance of two novel variants in DCM related genes is associated with a severe form of DCM. Exome sequencing has been shown to be very useful in identifying pathogenic mutations in cardiomyopathy families, and this report emphasizes the importance of comprehensive screening of DCM related genes, even after the identification of a single disease-causing mutation.
家族性扩张型心肌病(DCM)的特征是心室扩张和心肌功能减退。它是一种基因异质性疾病,与60多个基因的突变有关。我们对两名患病家庭成员进行了全外显子组测序,并结合心肌病相关基因筛选,以确定一个患有DCM的伊朗近亲家庭中可能的致病突变。在心肌病相关基因中鉴定出两个新的变异:肌钙蛋白T2型基因(TNNT2)中的c.247 A>C;p.N83H和肌球蛋白重链多肽7基因(MYH7)中的c.2863G>A;p.D955N。对其余家庭成员进行的桑格测序和共分离分析支持了这些双基因突变异在该家族患病成员中的共存。单独携带任何一种变异的个体均无症状。总之,我们发现DCM相关基因中两个新变异的双基因遗传与严重形式的DCM有关。外显子组测序已被证明在鉴定心肌病家族中的致病突变方面非常有用,本报告强调了全面筛查DCM相关基因的重要性,即使在鉴定出单一致病突变之后。