用体内、体外模型和临床样本综合阐明心肌病的蛋白质组和代谢组。

IF 12.1 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Molecular Therapy Pub Date : 2024-09-03 DOI:10.1016/j.ymthe.2024.08.030
Yiwei Hu, Yunzeng Zou, Liang Qiao, Ling Lin
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引用次数: 0

摘要

心肌病是一种常见的心血管疾病,影响各个年龄段的人,并可导致危及生命的心力衰竭。尽管心肌病有多种类型,每种类型都有不同的特征和病因,但我们对心肌病在系统生物学层面上的了解仍不全面。基于质谱的技术已成为强大的工具,可提供全面的分子图谱,并有助于发现生物标志物和阐明机理。本综述强调了将蛋白质组学和代谢组学方法与专业数据库相结合以确定不同类型心肌病的生物标志物和治疗靶点的巨大潜力。体内和体外模型,如转基因小鼠、患者衍生干细胞或诱导多能干细胞以及器官芯片,对于探索这种疾病复杂的病理生理学具有宝贵的价值。通过将 omics 方法与这些复杂的建模系统相结合,可以大大提高我们对心肌病分子基础的理解,促进诊断标志物和治疗策略的开发。有希望的治疗靶点包括那些参与细胞外基质重塑、肌节损伤和代谢重塑的靶点。这些靶点为推进心肌病的精准治疗提供了潜力,为针对患者的特定分子特征进行更有效的治疗带来了希望。
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Integrative Proteomic and Metabolomic Elucidation of Cardiomyopathy with in vivo, in vitro Models and Clinical Samples.

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 mechanism. 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 modelling 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 potential for the advancement of precision therapy in cardiomyopathy, offering hope for more effective treatments tailored to the specific molecular profiles of patients.

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来源期刊
Molecular Therapy
Molecular Therapy 医学-生物工程与应用微生物
CiteScore
19.20
自引率
3.20%
发文量
357
审稿时长
3 months
期刊介绍: Molecular Therapy is the leading journal for research in gene transfer, vector development, stem cell manipulation, and therapeutic interventions. It covers a broad spectrum of topics including genetic and acquired disease correction, vaccine development, pre-clinical validation, safety/efficacy studies, and clinical trials. With a focus on advancing genetics, medicine, and biotechnology, Molecular Therapy publishes peer-reviewed research, reviews, and commentaries to showcase the latest advancements in the field. With an impressive impact factor of 12.4 in 2022, it continues to attract top-tier contributions.
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