高血压、动脉粥样硬化和家族性高胆固醇血症的基因疗法:旧观念与新时代

Biologics Pub Date : 2024-04-19 DOI:10.3390/biologics4020010
Nikolaos Evangelidis, P. Evangelidis
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引用次数: 0

摘要

在 21 世纪,心血管疾病仍然是导致死亡的主要原因。高血压、血管粥样硬化和家族性高胆固醇血症(FH)是导致患者死亡率和发病率上升的原因。心血管疾病的治疗方法以药物治疗为主,但在精准医疗时代,个性化治疗方法正在被开发出来。研究表明,这些疾病有很强的遗传背景,这为针对这些疾病实施基因疗法创造了机会。目前,基因疗法尚未广泛应用于临床实践。这一研究领域的最新进展使基因疗法成为一种非常有前景的心血管疾病预防和治疗工具。本质性高血压(EH)的病理生理学主要基于交感神经系统和肾素血管紧张素醛固酮系统(RAAS)的激活、钠利肽的产生以及内皮功能障碍。质粒 DNA 和病毒载体可用于针对 EH 发病的主要机制。多年来已开展了许多临床前研究,结果显示血压显著下降。然而,目前还没有关于在 EH 中使用基因疗法的临床研究。动脉粥样硬化损伤是由单基因疾病引起的,或因血管壁的炎症激活而恶化。针对脂蛋白和胆固醇代谢以及血管炎症的基因治疗研究已进入前期和临床阶段。高胆固醇血症是一种常见的遗传代谢疾病,与血液中的高胆固醇水平有关。基因疗法的临床试验已经开展,并取得了令人乐观的结果。本综述概述了基因疗法治疗心血管疾病所面临的挑战。尽管如此,还需要进行更多的临床试验,以便为我们的患者开发出方便、安全的药物方案。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Gene Therapy for Hypertension, Atherosclerosis, and Familial Hypercholesterolemia: The Old Concepts and the New Era
Cardiovascular disease remains the main cause of mortality in the 21st century. Hypertension, vessel atherosclerosis, and familial hypercholesterolemia (FH) are responsible for increased mortality and morbidity in patients. Therapies for cardiovascular disease are based on drug treatment options, but in the era of precision medicine, personalized treatments are being developed. Studies have shown that these conditions have a strong genetic background, creating an opportunity for the implementation of gene therapy for these diseases. Currently, gene therapy is not widely used in clinical practice. Recent advances in this research field are making gene therapy a very promising preventive and therapeutic tool for cardiovascular disease. Essential hypertension’s (EH) pathophysiology is mostly based on the activation of both the sympathetic nervous system and the renin angiotensin aldosterone system (RAAS), natriuretic peptide production, and endothelial dysfunction. Plasmid DNA and viral vectors can be used, targeting the main mechanisms in the pathogenesis of EH. Many preclinical studies have been developed across the years, presenting a significant decrease in blood pressure. Nevertheless, no clinical studies have been developed studying the implementation of gene therapy in EH. Atherosclerotic damage is caused by monogenic diseases or is deteriorated by the activation of inflammation in the vessel wall. Gene therapy studies have been developed in the pre- and clinical phases targeting the lipoprotein and cholesterol metabolism and the inflammation of the vessels. FH is a common inherited metabolic disease associated with high levels of cholesterol in the blood. Clinical trials of gene therapy have been developed and presented optimistic results. In this review, the challenges of gene therapy for cardiovascular disease are outlined. Nevertheless, more clinical trials are needed to be performed for the development of convenient and safe drug schemes for our patients.
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