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RNA-based therapeutics, including siRNAs, miRNAs, and ASOs, hold significant potential for targeting previously undruggable genes and diseases, but face major delivery challenges due to evolutionary barriers like lipid bilayers and cellular defenses. Recent advancements in RNA chemistry have improved the stability and delivery of these therapeutics, particularly through modifications like phosphorothioate backbones and targeted delivery systems. Successful examples include ASOs for CNS disorders and ongoing

Author

linglongbing2023

Language

EN