MOLECULAR GENETICS: Edited by Ali J. MarianRNA-based therapeutics in cardiovascular diseasePham, Tan Pháta,*; Kremer, Veerlea,b,*; Boon, Reinier A.a,c,dAuthor Information aDepartment of Physiology, Amsterdam Cardiovascular Sciences, Vrije Universiteit, Amsterdam UMC location Vumc bDepartment of Medical Biochemistry, Amsterdam Cardiovascular Sciences, Universiteit van Amsterdam, Amsterdam UMC location AMC cInstitute for Cardiovascular Regeneration, Centre for Molecular Medicine, Goethe University dGerman Center for Cardiovascular Research (DZHK), Frankfurt am Main, Germany Correspondence to Dr Reinier A. Boon, Department of Physiology, Amsterdam UMC, De Boelelaan 1108, 1081 HZ Amsterdam, the Netherlands. Tel: +49 69 6301 85397; e-mail: email@example.com Current Opinion in Cardiology: May 2020 - Volume 35 - Issue 3 - p 191-198 doi: 10.1097/HCO.0000000000000724 Buy Metrics Abstract Purpose of review Cardiovascular disease is the leading cause of death globally, with the number of deaths rising every year. Much effort has gone into development of new treatment strategies. Many RNA species have important regulatory functions in disease initiation and progression, providing interesting new treatment options. This review focuses on different classes of RNA-based therapeutics and provides examples of current clinical and preclinical studies. Current challenges that prevent clinical translation and possibilities to overcome them will be discussed. Recent findings Different RNA-based molecules have been developed, such as antisense oligos, microRNA mimics and small interfering RNAs. Modifications are used to prevent degradation and immune activation and improve affinity. Additionally, in order to improve delivery of the RNA molecules to the target tissues, viral or nonviral vectors can be used. Summary RNA-based therapy has been shown to be a promising new treatment strategy for different disorders. However, several challenges, such as delivery problems and low efficacy remain. Future research will likely focus on effective delivery to target tissues in order to improve efficacy and avoid harmful side-effects. Copyright © 2020 Wolters Kluwer Health, Inc. All rights reserved.