siRNA and mRNA Therapeutics: A Comprehensive Review of Mechanisms, Delivery Strategies, Clinical Applications, and Future Directions in Precision Pharmacology.
Small interfering RNA (siRNA) and messenger RNA (mRNA) therapeutics represent transformative approaches in precision pharmacology, enabling targeted gene silencing and protein expression, respectively. This review provides a comprehensive analysis of their molecular mechanisms, delivery strategies, clinical applications, and future directions. By leveraging RNA interference for siRNA and translational machinery for mRNA, these therapeutics address previously untreatable diseases, including genetic disorders, cancers, and infectious diseases. Advances in nanoparticle- based delivery systems, such as lipid nanoparticles, have overcome historical barriers like RNA instability and immune activation. Current clinical applications, including FDA-approved therapies, highlight their efficacy, while ongoing challenges, such as off-target effects and scalable production, are discussed. Future directions emphasize personalized medicine, combination therapies, and novel delivery platforms to enhance therapeutic precision and accessibility.