Innovative RNA-Targeted Therapeutics: Bridging Biology and Clinical Applications

The field of RNA-targeted therapeutics is rapidly evolving, offering groundbreaking opportunities for the treatment of a wide array of diseases characterized by disrupted RNA processing. Recent advances in RNA biology have revealed that precise modulation of RNA stability and function is crucial in the pathogenesis of both genetic disorders and infectious diseases. While the promise of RNA-based drugs, such as small-molecule inhibitors targeting specific RNA-modulating enzymes like deadenylases, has garnered considerable attention, challenges persist in translating these findings from the laboratory to the clinic. These challenges include effective identification of drug candidates, understanding off-target effects, and achieving tissue-specific delivery.Increasingly sophisticated tools—such as molecular docking and fluorescence-based assays—have enabled high-throughput screening and detailed mechanistic studies of RNA-targeted compounds. Notably, small-molecule modulators of RNA stability offer a novel therapeutic axis, with preclinical models demonstrating substantial efficacy in correcting aberrant gene expression. However, sustained debate surrounds the optimization of specificity, scalability of production, and integration with existing therapeutic regimens. A comprehensive, interdisciplinary approach is needed to address these gaps, including better alignment between RNA biology and clinical methodologies.This Research Topic aims to accelerate the development and application of RNA-targeted therapeutics by fostering dialogue between molecular biology, medicinal chemistry, and clinical research. By highlighting innovative drug discovery methods and elucidating mechanisms of RNA regulation, the goal is to advance adaptable therapeutic strategies with broad clinical relevance. Key questions include: How can small-molecule inhibitors of deadenylases be optimized for specificity and efficacy? What translational pathways will best bridge preclinical discovery with clinical utility in genetic and viral diseases?The scope of this Research Topic encompasses experimental, computational, and translational approaches to RNA-targeted drug development, with particular emphasis on the interface between foundational science and therapeutic innovation. We welcome contributions that address, but are not limited to, the following themes:o Discovery and optimization of small-molecule inhibitors targeting RNA-modifying enzymeso High-throughput screening methodologies for RNA-targeted compoundso Mechanisms regulating RNA stability and their implications for therapeutic interventiono Preclinical and clinical evaluation of RNA-modulating drugs in disease modelso Integration of computational and experimental approaches in RNA-based drug designo Emerging strategies for delivery and specificity of RNA-targeted therapeutics Topic editors: Angelo Delmonte, Sarbjeet Makkar, POONAM RAMSEVAK PANDEY Source: https://www.frontiersin.org/research-topics/79373/innovative-rna-targeted-therapeutics-bridging-biology-and-clinical-applications