Department of Integrative Biology and Physiology, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
J Mol Cell Cardiol. 2012 Sep;53(3):446-57. doi: 10.1016/j.yjmcc.2012.05.018. Epub 2012 Jun 6.
Restrictive cardiomyopathy (RCM) has been linked to mutations in the thin filament regulatory protein cardiac troponin I (cTnI). As the pathogenesis of RCM from genotype to clinical phenotype is not fully understood, transgenic (Tg) mice were generated with cardiac specific expression of an RCM-linked missense mutation (R193H) in cTnI. R193H Tg mouse hearts with 15% stoichiometric replacement had smaller hearts and significantly elevated end diastolic pressures (EDP) in vivo. Using a unique carbon microfiber-based assay, membrane intact R193H adult cardiac myocytes generated higher passive tensions across a range of physiologic sarcomere lengths resulting in significant Ca(2+) independent cellular diastolic tone that was manifest in vivo as elevated organ-level EDP. Sarcomere relaxation and Ca(2+) decay was uncoupled in isolated R193H Tg adult myocytes due to the increase in myofilament Ca(2+) sensitivity of tension, decreased passive compliance of the sarcomere, and adaptive in vivo changes in which phospholamban (PLN) content was decreased. Further evidence of Ca(2+) and mechanical uncoupling in R193H Tg myocytes was demonstrated by the biphasic response of relaxation to increased pacing frequency versus the negative staircase seen with Ca(2+) decay. In comparison, non-transgenic myocyte relaxation closely paralleled the accelerated Ca(2+) decay. Ca(2+) transient amplitude was also significantly blunted in R193H Tg myocytes despite normal mechanical shortening resulting in myocyte hypercontractility when compared to non-transgenics. These results identify for the first time that a single point mutation in cTnI, R193H, directly causes elevated EDP due to a myocyte intrinsic loss of compliance independent of Ca(2+) cycling or altered cardiac morphology. The compound influence of impaired relaxation and elevated EDP represents a clinically severe form of diastolic dysfunction similar to the hemodynamic state documented in RCM patients.
限制型心肌病(RCM)与细肌丝调节蛋白心肌肌钙蛋白 I(cTnI)的突变有关。由于 RCM 从基因型到临床表型的发病机制尚未完全阐明,因此生成了具有心脏特异性表达 RCM 相关错义突变(R193H)的转基因(Tg)小鼠。cTnI 中的 R193H Tg 小鼠心脏有 15%的化学计量替换,心脏体积更小,体内舒张末期压力(EDP)显著升高。使用独特的碳纤维微纤维基检测,完整膜 R193H 成年心肌细胞在一系列生理肌节长度下产生更高的被动张力,导致显著的 Ca2+ 非依赖性细胞舒张张力,在体内表现为升高的器官水平 EDP。由于肌球蛋白丝 Ca2+ 敏感性张力增加、肌节的被动顺应性降低以及 PLN 含量减少等体内适应性变化,导致 R193H Tg 成年心肌细胞的肌节弛豫和 Ca2+ 衰减脱偶联。在 R193H Tg 心肌细胞中,通过增加肌球蛋白丝 Ca2+ 敏感性、弛豫对增加起搏频率的双相反应与 Ca2+ 衰减时的负阶梯相比,进一步证明了 Ca2+ 和机械解偶联的存在。相比之下,非转基因心肌细胞的弛豫与加速的 Ca2+ 衰减非常接近。尽管 R193H Tg 心肌细胞的机械缩短正常,但 Ca2+ 瞬变幅度也显著减弱,导致与非转基因相比心肌细胞的过度收缩。这些结果首次表明,cTnI 中的单个点突变 R193H 直接导致 EDP 升高,这是由于肌细胞内在顺应性丧失,与 Ca2+ 循环或心脏形态改变无关。舒张功能障碍的严重程度与 RCM 患者记录的血流动力学状态相似,舒张功能障碍的严重程度与松弛受损和 EDP 升高的复合影响有关。