从实验室到床边:rna疗法治疗高脂血症的最新进展

Yufei Zhou, Chen Chen
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引用次数: 1

摘要

周阿玉飞、陈晨*华中科技大学同济医学院同济医院心脏科、心脏科遗传与分子机制湖北省重点实验室,武汉430030*通信:chenchen@tjh.tjmu.edu.cn;电话和传真:86-27-6937-8422。摘要:高脂血症是构成代谢紊乱的疾病之一,是一种常见的公共卫生问题。这种疾病的特点是胆固醇、甘油三酯和/或脂蛋白水平升高;它被认为是许多疾病发病的危险因素,如2型糖尿病、非酒精性脂肪性肝病和心血管疾病。到目前为止,治疗高脂血症的主要药物是他汀类药物和抗枯草素/克辛9型蛋白转化酶(PCSK9)的单克隆抗体药物。他汀类药物长期使用的主要限制是难以忍受的副作用。Evolocumab和Alirocumab是两种针对PCSK9的单克隆抗体,可有效降低他汀类药物不耐受和家族性高胆固醇血症患者的低密度脂蛋白胆固醇(LDL-C)水平,且副作用较小。然而,这些单克隆抗体治疗由于半衰期短、费用高,可能导致患者不遵医嘱,给患者带来相当大的经济负担。鉴于RNA在基因调控中起着关键作用,基于RNA的治疗方法已成为设计新型抗高脂血症药物的有力蓝图。在此,我们总结了基于RNA的治疗策略和目前用于治疗高脂血症的RNA药物的临床试验。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
From Bench to Bedside: Current Developments in RNA-Based Therapies for Treatment of Hyperlipidemia
Review From Bench to Bedside: Current Developments in RNA-Based Therapies for Treatment of Hyperlipidemia Yufei Zhou and Chen Chen * Division of Cardiology and Hubei Key Laboratory of Genetics and Molecular Mechanisms of Cardiological Disorders, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, China. * Correspondence: chenchen@tjh.tjmu.edu.cn; Tel. & Fax: 86-27-6937-8422.     Received: 7 October 2022 Accepted: 1 November 2022 Published: 21 December 2022   Abstract: Hyperlipidemia is one of the conditions that constitute metabolic disorder and it is a common public health problem. The condition is characterized by increased levels of cholesterol, triglycerides and/or lipoproteins; it is a recognized as a risk factor for the onset of many diseases such as type 2 diabetes, non-alcoholic fatty liver disease, and cardiovascular disease. Up to now, the primary drugs for treating hyperlipidemia are statins and monoclonal antibody drugs against proprotein convertase subtilisin/kexin type 9 (PCSK9). The main limitation of statins for long-term use is intolerable side effects. Evolocumab and Alirocumab, two monoclonal antibodies against PCSK9, can effectively decrease the level of low-density lipoprotein cholesterol (LDL-C) in patients with statin intolerance and familial hypercholesterolemia, while causing fewer side effects. However, due to its short half-life and high costs, these monoclonal antibody treatments might result in patients’ non-compliance with medication and considerable economic burden on patients. Given that RNA plays a key role in gene regulation, RNA-based therapeutics have become powerful blueprints for designing new anti-hyperlipidemia drugs. Here, we summarized RNA-based therapeutic strategies and the current clinical trials for RNA drugs in hyperlipidemia treatment.
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