State Key Laboratory of Cardiovascular Disease, Beijing Key Laboratory for Molecular Diagnostics of Cardiovascular Diseases, Fuwai Hospital, National Center for Cardiovascular Diseases, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, People's Republic of China.
Departments of Cardiovascular Surgery, State Key Laboratory of Cardiovascular Disease, Fuwai Hospital, National Center for Cardiovascular Diseases, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, People's Republic of China.
Mol Genet Genomics. 2019 Oct;294(5):1241-1249. doi: 10.1007/s00438-019-01578-4. Epub 2019 May 18.
Hypertrophic cardiomyopathy (HCM), a major cause of sudden death in youth, is largely affected by genetic factors. The R58Q mutation in the MYL2 gene was identified in some HCM patients and was considered as a deleterious HCM mutation. However, the passing of R58Q between generations along with HCM symptoms was observed only in small families with only two or three members; thus, whether R58Q is as deleterious as previously claimed remains questionable. Here, we reported a large four-generation Chinese family, and found that R58Q existed in all six members with HCM and two healthy juveniles who had not yet developed HCM yet, and presumably in three deceased members who suffered from sudden death. In addition, we also found that compared with other mutations, R58Q had a more severe effect on the cellular level. Therefore, we confirmed that R58Q could be passed from generation to generation along with HCM symptoms and that it was indeed a deleterious mutation for HCM. However, further study is needed to identify additional factors that may determine the various symptoms shown in different family members within the same family.
肥厚型心肌病(HCM)是年轻人猝死的主要原因,主要受遗传因素影响。MYL2 基因中的 R58Q 突变已在一些 HCM 患者中被发现,并被认为是一种有害的 HCM 突变。然而,只有在只有两三个成员的小型家庭中才观察到 R58Q 在代际之间传递并伴有 HCM 症状,因此,R58Q 是否像以前声称的那样有害仍值得怀疑。在这里,我们报道了一个大型的四代中国家族,发现 R58Q 存在于所有 6 名患有 HCM 的成员和 2 名尚未患有 HCM 的健康青少年中,推测也存在于 3 名患有猝死的已故成员中。此外,我们还发现与其他突变相比,R58Q 在细胞水平上的影响更为严重。因此,我们证实 R58Q 可以随着 HCM 症状代代相传,并且确实是 HCM 的有害突变。然而,需要进一步的研究来确定可能决定同一家庭中不同家庭成员表现出的各种症状的其他因素。