RNA 疗法：现状与未来方向

RNA-based therapeutics have enabled drug discovery and design by introducing a fundamentally different perspective from conventional therapeutics. RNAs are not only the upstream molecules of proteins (coding) but also functional components of an extensive and intricate regulatory network (noncoding) that helps govern biological processes and mechanisms. Recent advances in functional genomics have accelerated the identification of functional RNAs with well-defined roles and their use in diagnostic and therapeutic approaches, which was previously challenging. RNA molecules can dynamically regulate gene function through their sequence and/or structure. Thus, RNA therapeutics offer distinct strategies for both loss- and gain-of-function interventions. This review focuses on druggable functional RNAs and discusses current and emerging technologies, as well as the scope for their potential clinical utility and targeted treatment. It also highlights the most studied optimization strategies and delivery systems designed to enhance the efficacy and tissue-specific delivery of RNA therapeutics to improve clinical outcomes. Finally, we offer our understanding and perspective on the field. Recent developments in enhancing the stability of these molecules in vivo have further improved the therapeutic potential of RNA-based approaches.