A Review on RNA-Based Therapeutics: Mechanisms, Applications, and Future Prospects

RNA-based therapeutics have gained considerable attention in recent years due to their ability to regulate disease processes at the molecular level. Unlike traditional pharmacological agents that primarily interact with proteins, these therapies function by influencing gene expression and RNA translation pathways. Various RNA modalities, including messenger RNA (mRNA), small interfering RNA (siRNA), antisense oligonucleotides, and microRNA, have been explored for therapeutic applications. RNA-based medicines have demonstrated promising outcomes in the treatment of genetic disorders, cancer, viral infections, and metabolic diseases. Advances in chemical modification techniques and delivery systems such as lipid nanoparticles have significantly improved the stability, bioavailability, and safety of RNA molecules, thereby enhancing their clinical potential. However, certain limitations including off-target effects, immune stimulation, and efficient tissue-specific delivery remain major challenges. This review presents a concise overview of RNA-based therapeutic strategies, their mechanisms of action, advantages, and current limitations. Additionally, it highlights recent developments and future prospects of RNA therapeutics in modern drug discovery. With continued research and technological innovation, RNA-based therapies are expected to play an increasingly important role in personalized and precision medicine.