RNA Therapeutics: Bridging Discovery and Clinical Implementation

RNA-based therapeutics represents a groundbreaking class of pharmaceuticals that harness the diverse functions of RNA molecules for therapeutic intervention. Key approaches include ribozymes, RNA interference, CRISPR interference, and messenger RNAs. The clinical applications of these technologies have been catalyzed by advancements in chemical modifications and delivery approaches, such as ionizable lipid nanoparticles. One of the key advantages of RNA-based drugs is their ability to target virtually any genetic component within the cell, including those deemed undruggable by small drug molecules. This chapter offers an in-depth summary of current RNA technologies, including their targeting mechanisms, clinical progresses and the challenges involved in improving their delivery, efficacy, and safety.