“RNA Therapy 101”: The basics of RNA therapy in questions and answers

Czech version

Authors: Jan Novák ^1,2
Authors‘ workplace: II. interní klinika LF MU a FN U sv. Anny ¹; Lékařská fakulta Masarykovy Univerzity, Brno ²
Published in: AtheroRev 2025; 10(3): 167-173
Category: Reviews

Overview

Cardiovascular diseases (CVD) remain the leading cause of death in developed countries. To further reduce mortality and morbidity, prevention and consistent treatment of modifiable risk factors for atherosclerotic cardiovascular disease—such as dyslipidemia—appear to be essential. In 2013, the first RNA therapy for dyslipidemia (mipomersen targeting ApoB, in the USA) was introduced, and since then the development of new drugs in this category has continued. RNA therapeutics already play (e.g., inclisiran targeting PCSK9) and will continue to play an increasingly significant role not only in preventive cardiology. The aim of this article is to provide a basic overview and to describe as simply as possible the current state of knowledge regarding RNA therapeutics, focusing on ASOs and siRNAs in lipidology, for clinicians with limited experience in molecular biology.

Keywords:

RNA interference – PCSK9 – ASO – RNA therapy – siRNA

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