RNA-based therapeutics in cardiovascular disease

被引:12
|
作者
Tan Phat Pham [1 ]
Kremer, Veerle [1 ,2 ]
Boon, Reinier A. [1 ,3 ,4 ]
机构
[1] Vrije Univ, Dept Physiol, Amsterdam UMC Locat Vumc, Amsterdam Cardiovasc Sci, Amsterdam, Netherlands
[2] Univ Amsterdam, Dept Med Biochem, Amsterdam UMC Locat AMC, Amsterdam Cardiovasc Sci, Amsterdam, Netherlands
[3] Goethe Univ, Ctr Mol Med, Inst Cardiovasc Regenerat, Frankfurt, Germany
[4] German Ctr Cardiovasc Res DZHK, Frankfurt, Germany
关键词
cardiovascular; clinical trial; delivery; nanoparticle; RNA therapeutics; ANTISENSE OLIGONUCLEOTIDES; DELIVERY; SIRNA; INTERFERENCE; CHALLENGES; STRATEGIES; MICRORNAS; EXOSOMES; EFFICACY;
D O I
10.1097/HCO.0000000000000724
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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. 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.
引用
收藏
页码:191 / 198
页数:8
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