RNA therapeutics: current status and future directions

Authors

• Barbara Yang, University of Texas at El Paso
• Melina J. Sedano, The University of Texas Rio Grande Valley School of MedicineFollow
• Karla Perez, The University of Texas Rio Grande Valley School of Medicine
• Mahalakshmi Vijayaraghavan, University of Texas at El Paso
• Victoria Reid, Texas Tech University Health Sciences Center El Paso
• Murali M. Yallapu Dr., The University of Texas Rio Grande Valley School of MedicineFollow
• Subhash Chauhan, The University of Texas Rio Grande Valley School of MedicineFollow
• Subramanian Dhandayuthapani, The University of Texas Rio Grande Valley School of MedicineFollow
• Enrique I. Ramos, University of Texas at El Paso
• Shrikanth S. Gadad, The University of Texas Rio Grande Valley School of MedicineFollow

Document Type

Article

Publication Date

9-24-2026

Abstract

RNA-based therapeutics have enabled drug discovery and design by introducing a fundamentally different perspective from conventional therapeutics. RNAs are not only the upstream molecules of proteins (coding) but also functional components of an extensive and intricate regulatory network (noncoding) that helps govern biological processes and mechanisms. Recent advances in functional genomics have accelerated the identification of functional RNAs with well-defined roles and their use in diagnostic and therapeutic approaches, which was previously challenging. RNA molecules can dynamically regulate gene function through their sequence and/or structure. Thus, RNA therapeutics offer distinct strategies for both loss- and gain-of-function interventions. This review focuses on druggable functional RNAs and discusses current and emerging technologies, as well as the scope for their potential clinical utility and targeted treatment. It also highlights the most studied optimization strategies and delivery systems designed to enhance the efficacy and tissue-specific delivery of RNA therapeutics to improve clinical outcomes. Finally, we offer our understanding and perspective on the field. Recent developments in enhancing the stability of these molecules in vivo have further improved the therapeutic potential of RNA-based approaches.

Comments

This article is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License, which permits any non-commercial use, sharing, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if you modified the licensed material. 

Recommended Citation

Yang, B., Sedano, M. J., Perez, K., Vijayaraghavan, M., Reid, V. A., Yallapu, M. M., ... & Gadad, S. S. (2026). RNA therapeutics: current status and future directions. Signal Transduction and Targeted Therapy, 11(1), 409. https://doi.org/10.1038/s41392-026-03032-5

Creative Commons License

This work is licensed under a Creative Commons Attribution-NonCommercial-No Derivative Works 4.0 International License.

Publication Title

Signal Transduction and Targeted Therapy

DOI

10.1038/s41392-026-03032-5

Academic Level

faculty

Mentor/PI Department

Immunology and Microbiology