Ebenezer Oluwakemi, Oyebamiji Abel Kolawole, Olanlokun John Oludele, Tuszynski Jack A, Wong Gane Ka-Shu
Department of Physics, University of Alberta, Edmonton, AB T6G 2E1, Canada.
Department of Industrial Chemistry, University of Ilesa, Ilesa PMB 5089, Nigeria.
Int J Mol Sci. 2025 Apr 8;26(8):3456. doi: 10.3390/ijms26083456.
Small interfering RNA (siRNA) has been deemed a promising therapeutic method for treating diverse diseases. siRNA-based therapeutics provide a distinct mechanism of action by selectively targeting and silencing disease-causing genes at the post-transcriptional level. This paper provides an overview of the present state of siRNA-based therapeutics, highlighting their potential in different therapeutic areas. The first section of this review introduces the basic principles of siRNA technology, including its mechanism of action and delivery methods. Subsequently, we discuss the impediments associated with siRNA delivery and manufacturing development and the strategies for overcoming these obstacles. The clinical advancement of siRNA therapeutics in various disease areas, including cancer, genetic disorders, viral infections, and inflammatory diseases, is summarized. Lastly, we summarize the successes, failures, and lessons learned from the development of siRNAs. With advancements in delivery systems and improvements in target selection, the field of medicine can be revolutionized, and siRNA therapeutics can offer new treatment options for patients.
小干扰RNA(siRNA)已被视为一种治疗多种疾病的有前景的治疗方法。基于siRNA的疗法通过在转录后水平选择性靶向并沉默致病基因,提供了一种独特的作用机制。本文概述了基于siRNA的疗法的现状,突出了它们在不同治疗领域的潜力。本综述的第一部分介绍了siRNA技术的基本原理,包括其作用机制和递送方法。随后,我们讨论了与siRNA递送和制造开发相关的障碍以及克服这些障碍的策略。总结了siRNA疗法在包括癌症、遗传性疾病、病毒感染和炎症性疾病在内的各种疾病领域的临床进展。最后,我们总结了siRNA开发中的成功、失败及经验教训。随着递送系统的进步和靶点选择的改善,医学领域可能会发生变革,siRNA疗法可以为患者提供新的治疗选择。