心血管疾病(CVDs)是一个巨大的全球健康负担。要求创新的治疗方法。近年来,mRNA疗法已成为有效对抗CVD的有希望的策略。与传统的小分子药物不同,mRNA治疗剂能够通过将特定mRNA分子递送至靶细胞来直接调节细胞功能。这种方法提供了前所未有的优势,包括利用内源性细胞机制进行蛋白质合成的能力,从而允许精确控制基因表达而不插入基因组。这篇综述总结了在心血管疾病背景下细胞特异性mRNA疗法的潜力的现状。首先,它概述了与传统CVD治疗相关的挑战,并强调了靶向治疗的必要性.随后,它阐明了mRNA疗法的基本原理和先进的递送系统的发展,以确保细胞特异性和增强的疗效。值得注意的是,创新的递送方法,如脂质纳米粒和外泌体已显示出改善mRNA向心脏细胞的靶向递送的希望,活化成纤维细胞,和其他相关细胞类型。此外,该综述强调了细胞特异性mRNA疗法在解决心血管疾病各个方面的不同应用,包括动脉粥样硬化,心肌梗塞,心力衰竭,和心律失常.通过调节参与心肌细胞增殖的关键调控基因,炎症,血管生成,组织修复,和细胞存活,mRNA疗法具有在CVD发病机理的多个阶段进行干预的潜力。尽管潜力巨大,本摘要承认将细胞特异性mRNA疗法从临床前研究转化为临床应用如脱靶效应和递送的挑战.总之,细胞特异性mRNA疗法已成为CVD的革命性基因治疗方法,提供有针对性的干预措施,有可能显着改善患者的预后。
Cardiovascular diseases (CVDs) represent a significant global health burden, demanding innovative therapeutic approaches. In recent years, mRNA therapeutics have emerged as a promising strategy to combat CVDs effectively. Unlike conventional small-molecule drugs, mRNA therapeutics enable the direct modulation of cellular functions by delivering specific mRNA molecules to target cells. This approach offers unprecedented advantages, including the ability to harness endogenous cellular machinery for protein synthesis, thus allowing precise control over gene expression without insertion into the genome. This review summarizes the current status of the potential of cell-specific mRNA therapeutics in the context of cardiovascular diseases. First, it outlines the challenges associated with traditional CVD treatments and emphasizes the need for targeted therapies. Subsequently, it elucidates the underlying principles of mRNA therapeutics and the development of advanced delivery systems to ensure cell-specificity and enhanced efficacy. Notably, innovative delivery methods such as lipid nanoparticles and exosomes have shown promise in improving the targeted delivery of mRNA to cardiac cells, activated fibroblasts, and other relevant cell types. Furthermore, the review highlights the diverse applications of cell-specific mRNA therapeutics in addressing various aspects of cardiovascular diseases, including atherosclerosis, myocardial infarction, heart failure, and arrhythmias. By modulating key regulatory genes involved in cardiomyocyte proliferation, inflammation, angiogenesis, tissue repair, and cell survival, mRNA therapeutics hold the potential to intervene at multiple stages of CVD pathogenesis. Despite its immense potential, this abstract acknowledges the challenges in translating cell-specific mRNA therapeutics from preclinical studies to clinical applications like off-target effects and delivery. In conclusion, cell-specific mRNA therapeutics have emerged as a revolutionary gene therapy approach for CVD, offering targeted interventions with the potential to significantly improve patient outcomes.