Weisleder Noah, Taffet George E, Capetanaki Yassemi
Department of Molecular and Cellular Biology, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, USA.
Proc Natl Acad Sci U S A. 2004 Jan 20;101(3):769-74. doi: 10.1073/pnas.0303202101. Epub 2004 Jan 8.
One of the hallmarks of cardiomyopathy and heart failure is pronounced and progressive cardiomyocyte death. Understanding the mechanisms involved in cardiomyocyte cell death is a topic of great interest for treatment of cardiac disease. Mice null for desmin, the muscle-specific member of the intermediate filament gene family, develop cardiomyopathy characterized by extensive cardiomyocyte death, fibrosis, calcification, and eventual heart failure. The earliest ultrastructural defects are observed in mitochondria. In the present study, we have demonstrated that these mitochondrial abnormalities are the primary cause of the observed cardiomyopathy and that these defects can be ameliorated by overexpression of bcl-2 in desmin null heart. Overexpression of bcl-2 in the desmin null heart results in correction of mitochondrial defects, reduced occurrence of fibrotic lesions in the myocardium, prevention of cardiac hypertrophy, restoration of cardiomyocyte ultrastructure, and significant improvement of cardiac function. Furthermore, we have found that loss of desmin also diminishes the capacity of mitochondria to resist exposure to calcium, a defect that can be partially restored by bcl-2 overexpression. These results point to a unique function for desmin in protection of mitochondria from calcium exposure that can be partially rescued by overexpression of bcl-2. We show that bcl-2 cardiac overexpression has provided significant improvement of an inherited form of cardiomyopathy, revealing the potential for bcl-2, and perhaps other genes in the family, as therapeutic agents for heart disease of many types, including inherited forms.
心肌病和心力衰竭的一个标志是明显且进行性的心肌细胞死亡。了解心肌细胞死亡所涉及的机制是治疗心脏病的一个备受关注的话题。结蛋白基因缺失的小鼠,结蛋白是中间丝基因家族的肌肉特异性成员,会发展出以广泛心肌细胞死亡、纤维化、钙化以及最终心力衰竭为特征的心肌病。最早的超微结构缺陷在线粒体中被观察到。在本研究中,我们已经证明这些线粒体异常是所观察到的心肌病的主要原因,并且这些缺陷可以通过在结蛋白基因缺失的心脏中过表达bcl-2来改善。在结蛋白基因缺失的心脏中过表达bcl-2会导致线粒体缺陷的纠正、心肌纤维化病变发生率的降低、心脏肥大的预防、心肌细胞超微结构的恢复以及心脏功能的显著改善。此外,我们发现结蛋白的缺失也会降低线粒体抵抗钙暴露的能力,这种缺陷可以通过bcl-2的过表达部分恢复。这些结果表明结蛋白在保护线粒体免受钙暴露方面具有独特功能,这种功能可以通过bcl-2的过表达部分挽救。我们表明心脏过表达bcl-2已经显著改善了一种遗传性心肌病的症状,揭示了bcl-2以及该家族中其他基因作为多种类型心脏病(包括遗传性心脏病)治疗药物的潜力。