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RNA therapeutics in cardiovascular medicine
被引:0
|作者:
Damase, Tulsi Ram
[1
]
Cooke, John P.
[1
]
机构:
[1] Houston Methodist Acad Inst, Ctr RNA Therapeut, Dept Cardiovasc Sci, 6670 Bertner Ave, Houston, TX 77030 USA
基金:
美国国家卫生研究院;
关键词:
antisense oligonucleotide;
cardiovascular medicine;
messenger RNA;
microRNA;
RNA therapeutics;
small interfering RNA;
MESSENGER-RNA;
FAMILIAL HYPERCHOLESTEROLEMIA;
TARGETING PCSK9;
GENE-TRANSFER;
TRANSLATION;
EFFICACY;
THERAPY;
ACID;
INTERFERENCE;
ETEPLIRSEN;
D O I:
10.1097/HCO.0000000000001210
中图分类号:
R5 [内科学];
学科分类号:
1002 ;
100201 ;
摘要:
Purpose of reviewRNA 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 findingsThe 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.SummaryBecause 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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页码:139 / 149
页数:11
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