University Centre of Excellence in Research, Baba Farid University of Health Sciences, Faridkot, India.
Multidisciplinary Research Unit, Guru Gobind Singh Medical College, Faridkot, India.
Adv Exp Med Biol. 2020;1229:369-381. doi: 10.1007/978-981-15-1671-9_23.
Ribonucleic acid (RNA) is being exploited and understood in its many aspects of function and structure for development of valuable tools in the therapeutics of various diseases such as cardiovascular etc. The expanded knowledge regarding function of RNA in the genomics and inside the cell has dramatically changed the therapeutic strategies in the past few years. RNA has become a spotlight of attention for developing novel therapeutic schemes and hence variety of therapeutic strategies is being coming into the picture that includes RNA interference, use of aptamers, role of microRNA (miRNA) that can alter the complex gene expression patterns. It is due to the fact that RNA offers various advantages in disease management as it can be edited and modified in its various forms such as secondary and tertiary structures. Although scientists are in process of manufacturing RNA-targeting therapies using variety of endogenous gene silencing regulators, Small interfering RNAs (Si RNAs), aptamers and microRNA for cardiovascular diseases yet the development of a novel, risk free therapeutic strategy is a major challenge and need of the hour in cardiovascular medicine. In this regard these agents are required to overcome pleothra of barriers such as stability of drug targets, immunogenicity, adequate binding, targeted delivery etc. to become effective drugs. Recent years have witnessed the progress of RNA therapeutic strategies in cardiovascular diseases that are likely to significantly expand the cardiovascular therapeutic repertoire within the next decade. The present manuscript has been compiled to summarize various approaches of siRNA based therapies in cardiovascular diseases along with the advantages, outcomes and limitations if any in this regard. In addition, the future prospects of RNA therapeutic modalities in cardiovascular diseases are summarized.
核糖核酸(RNA)在其功能和结构的许多方面都在被开发和理解,以便在治疗各种疾病(如心血管疾病等)方面开发有价值的工具。关于 RNA 在基因组学和细胞内的功能的扩展知识在过去几年中极大地改变了治疗策略。RNA 已成为开发新治疗方案的焦点,因此各种治疗策略正在出现,包括 RNA 干扰、适体的使用、微小 RNA(miRNA)的作用,这些都可以改变复杂的基因表达模式。这是因为 RNA 在疾病管理方面提供了各种优势,因为它可以在各种形式(如二级和三级结构)中进行编辑和修饰。尽管科学家们正在使用各种内源性基因沉默调节剂、小干扰 RNA(siRNA)、适体和 miRNA 来制造针对心血管疾病的 RNA 靶向治疗方法,但开发一种新型、无风险的治疗策略是心血管医学的主要挑战和当务之急。在这方面,这些药物需要克服药物靶点稳定性、免疫原性、充分结合、靶向递送等多方面的障碍,才能成为有效的药物。近年来,RNA 治疗策略在心血管疾病方面取得了进展,有望在未来十年内显著扩大心血管治疗范围。本综述旨在总结基于 siRNA 的心血管疾病治疗方法的各种途径,以及在这方面的优势、结果和局限性。此外,还总结了 RNA 治疗模式在心血管疾病中的未来前景。