Rasooly Danielle, Pereira Alexandre C, Joseph Jacob
Massachusetts Veterans Epidemiology Research and Information Collaborative (MAVERIC), Veterans Affairs Healthcare System, 150 S. Huntington Ave., Boston, MA 02130, USA.
Division of Aging, Brigham and Women's Hospital, Harvard Medical School, 75 Francis St., Boston, MA 02130, USA.
Int J Mol Sci. 2025 Mar 17;26(6):2703. doi: 10.3390/ijms26062703.
Heart failure (HF) is a complex, heterogeneous syndrome with rising prevalence and high morbidity and mortality. The pathophysiology and diverse etiologies of HF present significant challenges for developing effective therapies. Omics technologies-including genomics, proteomics, transcriptomics, metabolomics, and epigenomics-have reshaped our understanding of HF at the molecular level, uncovering new biomarkers and potential therapeutic targets. Omics also enable insights into individualized treatment responses, the risks of adverse drug effects, and patient stratification for clinical trials. This review explores how multi-omics can enhance heart failure drug discovery and development across all stages of the therapeutic pipeline: (1) target selection and lead identification, (2) preclinical studies, and (3) clinical trials. By integrating omics approaches throughout the drug development process, we can accelerate the discovery of more effective and personalized therapies for heart failure.
心力衰竭（HF）是一种复杂的异质性综合征，其患病率不断上升，发病率和死亡率都很高。HF的病理生理学和多种病因给开发有效治疗方法带来了重大挑战。组学技术——包括基因组学、蛋白质组学、转录组学、代谢组学和表观基因组学——在分子水平上重塑了我们对HF的理解，发现了新的生物标志物和潜在的治疗靶点。组学还能够深入了解个体化治疗反应、药物不良反应风险以及临床试验中的患者分层。本综述探讨了多组学如何在治疗管道的各个阶段加强心力衰竭药物的发现和开发：（1）靶点选择和先导物识别，（2）临床前研究，以及（3）临床试验。通过在整个药物开发过程中整合组学方法，我们可以加速发现更有效、更个性化的心力衰竭治疗方法。
