Department of Chemistry, Zhongshan Hospital, and Minhang Hospital, Fudan University, Shanghai 200000, China.
Department of Chemistry, Zhongshan Hospital, and Minhang Hospital, Fudan University, Shanghai 200000, China.
Mol Ther. 2024 Oct 2;32(10):3288-3312. doi: 10.1016/j.ymthe.2024.08.030. Epub 2024 Sep 3.
Cardiomyopathy is a prevalent cardiovascular disease that affects individuals of all ages and can lead to life-threatening heart failure. Despite its variety in types, each with distinct characteristics and causes, our understanding of cardiomyopathy at a systematic biology level remains incomplete. Mass spectrometry-based techniques have emerged as powerful tools, providing a comprehensive view of the molecular landscape and aiding in the discovery of biomarkers and elucidation of mechanisms. This review highlights the significant potential of integrating proteomic and metabolomic approaches with specialized databases to identify biomarkers and therapeutic targets across different types of cardiomyopathies. In vivo and in vitro models, such as genetically modified mice, patient-derived or induced pluripotent stem cells, and organ chips, are invaluable in exploring the pathophysiological complexities of this disease. By integrating omics approaches with these sophisticated modeling systems, our comprehension of the molecular underpinnings of cardiomyopathy can be greatly enhanced, facilitating the development of diagnostic markers and therapeutic strategies. Among the promising therapeutic targets are those involved in extracellular matrix remodeling, sarcomere damage, and metabolic remodeling. These targets hold the potential to advance precision therapy in cardiomyopathy, offering hope for more effective treatments tailored to the specific molecular profiles of patients.
心肌病是一种常见的心血管疾病，可影响各个年龄段的人群，并可导致危及生命的心力衰竭。尽管心肌病有多种类型，每种类型都有其独特的特征和病因，但我们对系统性生物学水平上的心肌病的理解仍不完整。基于质谱的技术已成为强大的工具，提供了对分子景观的全面了解，并有助于发现生物标志物和阐明机制。这篇综述强调了将蛋白质组学和代谢组学方法与专门的数据库相结合，以识别不同类型心肌病中的生物标志物和治疗靶点的巨大潜力。体内和体外模型，如基因修饰小鼠、患者来源或诱导多能干细胞以及器官芯片，在探索这种疾病的病理生理复杂性方面具有重要价值。通过将组学方法与这些复杂的建模系统相结合，可以大大增强我们对心肌病分子基础的理解，从而促进诊断标志物和治疗策略的发展。有前途的治疗靶点包括涉及细胞外基质重塑、肌节损伤和代谢重塑的靶点。这些靶点有可能推进心肌病的精准治疗，为针对患者特定分子谱的更有效治疗方法带来希望。
