医学
核糖核酸
临床试验
疾病
反义治疗
食品药品监督管理局
药品
小干扰RNA
药理学
生物信息学
RNA干扰
重症监护医学
内科学
基因
生物
生物化学
锁核酸
作者
Tulsi Ram Damase,John P. Cooke
标识
DOI:10.1097/hco.0000000000001210
摘要
Purpose of review RNA therapeutics came to global attention when mRNA-based vaccines provided an answer to the SARS-CoV-2 pandemic. The immense significance of this development notwithstanding, it is important to note that almost a decade prior to the pandemic, RNA drugs had made important inroads toward the amelioration of disease. The first class of RNA therapies to be introduced into clinical use were the antisense oligomers and siRNA drugs which generally induce a therapeutic effect by acting to brake or to modulate mRNA expression. RNA therapeutics is quickly becoming the fourth pillar of pharmacotherapy, and will have broad applications, including for the treatment of cardiovascular disease. Recent findings The United States (US) Food and Drug Administration (FDA) has approved several antisense oligomers (ASOs) and siRNA-based drugs to treat disorders associated with cardiovascular disease. In addition, multiple RNA-based drugs are in clinical trials to assess their safety and efficacy in patients with cardiovascular disorders, such as Zodasiran, a siRNA therapy that targets angiopoietin-like protein 3 (ANGPTL3) to reduce LDL cholesterol. Summary Because of limitless sequence choice; speed of design; and relative ease of synthesis, RNA drugs will be rapidly developed, will have broad applications, and will be generated at lower cost than other drug types. This review aims to highlight RNA therapies for cardiovascular diseases that are approved, and those that are under clinical evaluation.
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