Emory University School of Medicine, Atlanta, Georgia, USA.
Harvard Medical School, Boston, Massachusetts, USA.
JCI Insight. 2020 Aug 6;5(15):138687. doi: 10.1172/jci.insight.138687.
The bromodomain and extraterminal (BET) family comprises epigenetic reader proteins that are important regulators of inflammatory and hypertrophic gene expression in the heart. We previously identified the activation of proinflammatory gene networks as a key early driver of dilated cardiomyopathy (DCM) in transgenic mice expressing a mutant form of phospholamban (PLNR9C) - a genetic cause of DCM in humans. We hypothesized that BETs coactivate this inflammatory process, representing a critical node in the progression of DCM. To test this hypothesis, we treated PLNR9C or age-matched WT mice longitudinally with the small molecule BET bromodomain inhibitor JQ1 or vehicle. BET inhibition abrogated adverse cardiac remodeling, reduced cardiac fibrosis, and prolonged survival in PLNR9C mice by inhibiting expression of proinflammatory gene networks at all stages of disease. Specifically, JQ1 had profound effects on proinflammatory gene network expression in cardiac fibroblasts, while having little effect on gene expression in cardiomyocytes. Cardiac fibroblast proliferation was also substantially reduced by JQ1. Mechanistically, we demonstrated that BRD4 serves as a direct and essential regulator of NF-κB-mediated proinflammatory gene expression in cardiac fibroblasts. Suppressing proinflammatory gene expression via BET bromodomain inhibition could be a novel therapeutic strategy for chronic DCM in humans.
溴结构域和末端(BET)家族包含表观遗传读取蛋白,它们是心脏中炎症和肥大基因表达的重要调节剂。我们之前发现,在表达突变型肌浆球蛋白结合蛋白 C(PLNR9C)的转基因小鼠中,促炎基因网络的激活是扩张型心肌病(DCM)的一个关键早期驱动因素,PLNR9C 是人类 DCM 的一种遗传原因。我们假设 BET 共同激活了这一炎症过程,这代表着 DCM 进展中的一个关键节点。为了验证这一假设,我们对 PLNR9C 或年龄匹配的 WT 小鼠进行了纵向治疗,使用小分子 BET 溴结构域抑制剂 JQ1 或载体。BET 抑制通过在疾病的所有阶段抑制促炎基因网络的表达,消除了 PLNR9C 小鼠的不良心脏重塑、减少了心脏纤维化并延长了其生存时间。具体来说,JQ1 对心肌成纤维细胞中促炎基因网络的表达有深远影响,而对心肌细胞中的基因表达影响很小。JQ1 还大大减少了心肌成纤维细胞的增殖。从机制上讲,我们证明 BRD4 是心肌成纤维细胞中 NF-κB 介导的促炎基因表达的直接和必需调节剂。通过 BET 溴结构域抑制抑制促炎基因表达可能是人类慢性 DCM 的一种新的治疗策略。