Abstract:
RNA-based vaccines have sparked a paradigm shift in the treatment and prevention of diseases by nucleic acid medicines. There has been a notable surge in the development of nucleic acid therapeutics and vaccines following the global approval of the two messenger RNA-based COVID-19 vaccines. This growth is fueled by the exploration of numerous RNA products in preclinical stages, offering several advantages over conventional methods, i.e., safety, efficacy, scalability, and cost-effectiveness. In this chapter, we provide an overview of various types of RNA and their mechanisms of action for stimulating immune responses and inducing therapeutic effects. Furthermore, this chapter delves into the varying delivery systems, particularly emphasizing the use of nanoparticles to deliver RNA. The choice of delivery system is an intricate process involved in developing nucleic acid medicines that significantly enhances their stability, biocompatibility, and site-specificity. Additionally, this chapter sheds light on the current landscape of clinical trials of RNA therapeutics and vaccines against intracellular pathogens.
摘要:
基于RNA的疫苗引发了核酸药物治疗和预防疾病的范式转变。在两种基于信使RNA的COVID-19疫苗获得全球批准后,核酸疗法和疫苗的开发出现了显著的激增。这种增长是由临床前阶段对许多RNA产物的探索推动的,提供了优于传统方法的几个优点,即,安全,功效,可扩展性,和成本效益。在这一章中,我们概述了各种类型的RNA及其刺激免疫反应和诱导治疗效果的作用机制。此外,本章深入研究了不同的交付系统,特别强调使用纳米颗粒递送RNA。递送系统的选择是一个复杂的过程,涉及开发核酸药物,显着增强其稳定性,生物相容性,和位点特异性。此外,这一章揭示了针对细胞内病原体的RNA疗法和疫苗的临床试验的现状。