Guillermo Tramontin and Cecilia Piergentili
While traditionally small molecules medicines and antibodies target specific proteins, RNA therapeutics target RNA molecules involved in disease processes, allowing for more precise and effective treatment.
A key advantage of RNA therapeutics is the ability to target previously "undruggable" targets, such as transcription factors and non-coding RNAs, which are often involved in complex disease pathways.
Additionally, RNA-based treatments can be designed to be highly selective and specific, reducing the risk of off-target effects and improving safety profiles (Winkler, 2021; Zhu et al. 2022).
Development of these innovative therapies has been facilitated by technological advances and an understanding of RNA biology, including synthesizing and delivery of RNA molecules to specific tissues and cells in the body.