Jeong Jiwon, Jeong Sunjoo
Laboratory of RNA Cell Biology, Department of Bioconvergence Engineering, Dankook University, Yongin, Republic of Korea.
Anim Cells Syst (Seoul). 2025 Aug 25;29(1):523-543. doi: 10.1080/19768354.2025.2548253. eCollection 2025.
Breakthrough discoveries in RNA biology have led to a paradigm shift in our understanding of RNA-from passive intermediates to active regulators of gene expression. Technological innovations and deeper insights into RNA regulation have transformed the field, positioning RNA as a powerful tool for therapeutic development. Recent advances in RNA technologies have revolutionized medicine by enabling the precise targeting of specific mRNAs to modulate aberrant transcripts, correct genetic defects, and reprogram cellular behavior. This review provides an overview of the coordinated regulation of mRNA processing and its application to RNA-based therapeutics, including antisense oligonucleotides (ASOs), splice-switching oligonucleotides (SSOs), small interfering RNAs (siRNAs), and Aptamers. We focus on clinically approved RNA therapeutics, emphasizing their biological mechanisms such as RNA stability and splicing regulation. The expanding repertoire of RNA technologies underscores the translational potential of RNA biology and its growing clinical impact. Future developments are expected to yield highly specific, modular, and programmable RNA medicines capable of treating a wide range of previously intractable diseases.
RNA生物学的突破性发现使我们对RNA的理解发生了范式转变——从基因表达的被动中间体转变为主动调节因子。技术创新以及对RNA调控更深入的认识改变了这一领域，使RNA成为治疗开发的有力工具。RNA技术的最新进展通过实现对特定mRNA的精确靶向，来调节异常转录本、纠正基因缺陷和重新编程细胞行为，从而彻底改变了医学。本综述概述了mRNA加工的协同调控及其在基于RNA的治疗中的应用，包括反义寡核苷酸（ASO）、剪接转换寡核苷酸（SSO）、小干扰RNA（siRNA）和适配体。我们关注临床批准的RNA疗法，强调其生物学机制，如RNA稳定性和剪接调控。不断扩展的RNA技术库凸显了RNA生物学的转化潜力及其日益增长的临床影响。未来的发展有望产生能够治疗多种以前难以治疗的疾病的高度特异性、模块化和可编程的RNA药物。
