Murdoch Childrens Research Institute, Royal Children's Hospital, Flemington Road, Parkville, VIC, 3052, Australia.
The Florey Institute of Neuroscience and Mental Health; Centre for Neuroscience, University of Melbourne, Parkville, VIC, 3052, Australia.
Nat Commun. 2018 Apr 10;9(1):1373. doi: 10.1038/s41467-018-03714-x.
Congenital heart defects can be caused by mutations in genes that guide cardiac lineage formation. Here, we show deletion of NKX2-5, a critical component of the cardiac gene regulatory network, in human embryonic stem cells (hESCs), results in impaired cardiomyogenesis, failure to activate VCAM1 and to downregulate the progenitor marker PDGFRα. Furthermore, NKX2-5 null cardiomyocytes have abnormal physiology, with asynchronous contractions and altered action potentials. Molecular profiling and genetic rescue experiments demonstrate that the bHLH protein HEY2 is a key mediator of NKX2-5 function during human cardiomyogenesis. These findings identify HEY2 as a novel component of the NKX2-5 cardiac transcriptional network, providing tangible evidence that hESC models can decipher the complex pathways that regulate early stage human heart development. These data provide a human context for the evaluation of pathogenic mutations in congenital heart disease.
先天性心脏缺陷可能是由指导心脏谱系形成的基因突变引起的。在这里,我们显示 NKX2-5 的缺失,即心脏基因调控网络的关键组成部分,在人类胚胎干细胞(hESC)中导致心肌发生受损,无法激活 VCAM1 并下调祖细胞标志物 PDGFRα。此外,NKX2-5 缺失的心肌细胞具有异常的生理学特性,表现为收缩不同步和动作电位改变。分子谱分析和遗传挽救实验表明,bHLH 蛋白 HEY2 是人类心肌发生过程中 NKX2-5 功能的关键介质。这些发现将 HEY2 确定为 NKX2-5 心脏转录网络的一个新组成部分,为 hESC 模型能够解析调节早期人类心脏发育的复杂途径提供了确凿的证据。这些数据为评估先天性心脏病中的致病突变提供了人类背景。