Department of Physiology, Amsterdam Cardiovascular Sciences, Vrije Universiteit, Amsterdam UMC location Vumc.
Department of Medical Biochemistry, Amsterdam Cardiovascular Sciences, Universiteit van Amsterdam, Amsterdam UMC location AMC.
Curr Opin Cardiol. 2020 May;35(3):191-198. doi: 10.1097/HCO.0000000000000724.
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.
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.
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.
心血管疾病是全球范围内导致死亡的主要原因，且每年的死亡人数都在不断增加。人们付出了大量努力来开发新的治疗策略。许多 RNA 种类在疾病的发生和发展中具有重要的调节功能，为新的治疗方法提供了有趣的选择。本综述重点关注基于 RNA 的不同治疗类别，并提供当前临床前和临床研究的实例。本文还将讨论当前阻止其临床转化的挑战和可能的解决方法。
已经开发出了不同的基于 RNA 的分子，例如反义寡核苷酸、microRNA 模拟物和小干扰 RNA。通过修饰来防止降解和免疫激活，并提高亲和力。此外，为了提高 RNA 分子向靶组织的递送效率，可以使用病毒或非病毒载体。
基于 RNA 的治疗已被证明是一种有前途的新的治疗方法，可用于多种疾病。然而，仍存在一些挑战，如递送问题和低疗效。未来的研究可能集中在有效递送到靶组织，以提高疗效并避免有害的副作用。
