Unveiling the potential of RNA-targeting therapies

RNA-targeting therapies, particularly antisense oligonucleotides (ASOs), have emerged as a promising frontier in molecular medicine. This review explores the potential and challenges of these therapies, highlighting their unique advantages such as design flexibility, broad target range, and high specificity. We discuss the critical role of RNA structure in ASO design and efficacy, emphasizing the need for advanced computational tools in structure prediction and analysis. Recent advancements in artificial intelligence and machine learning are shown to significantly improve ASO design and toxicity prediction. While several ASO therapies have gained approval, we also examine the hurdles faced in clinical trials, including delivery challenges, toxicity concerns, and off-target effects. The paper concludes that the integration of cutting-edge computational methods, enhanced chemical modifications, and innovative delivery strategies is paving the way for more effective and safer ASO therapies, with an increasing number of RNA-targeting therapies expected to transition from laboratory to clinic as our understanding of RNA biology deepens and technologies evolve.

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