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Review
. 2025 May 5:947:149342.
doi: 10.1016/j.gene.2025.149342. Epub 2025 Feb 19.

RNA research for drug discovery: Recent advances and critical insight

Affiliations
Review

RNA research for drug discovery: Recent advances and critical insight

Patrick Maduabuchi Aja et al. Gene. .

Abstract

The field of RNA research has experienced significant changes and is now at the forefront of contemporary drug development. This narrative overview explores the scientific developments and historical turning points in RNA research, emphasising the field's critical significance in the development of novel therapeutics. Important discoveries like antisense oligonucleotides (ASOs), mRNA therapies, and RNA interference (RNAi) have created novel treatment options that can be targeted, such as the ground-breaking mRNA vaccinations against COVID-19. Advances in high-throughput sequencing, single-cell RNA sequencing, and epitranscriptomics have further unravelled the complexity of RNA biology, shedding light on the intricacies of gene regulation and cellular diversity. The integration of computational tools and bioinformatics has propelled the identification of RNA-based biomarkers and the development of RNA therapeutics. Despite significant progress, challenges such as RNA stability, delivery, and off-target effects persist, necessitating continuous innovation and ethical considerations. This review provides a critical insight into the current state and prospects of RNA research, emphasising its transformative potential in drug discovery. By examining the interplay between technological advancements and therapeutic applications, we underscore the promising horizon for RNA-based interventions in treating a myriad of diseases, marking a new era in precision medicine.

Keywords: Drug Discovery; RNA Research; RNA Technologies; RNA Therapeutics; Transcriptomics.

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Conflict of interest statement

Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: [Patrick Maduabuchi Aja reports was provided by Kampala International University - Western Campus. Patrick Maduabuchi Aja reports a relationship with Kampala International University - Western Campus that includes: employment. Patrick Maduabuchi Aja has patent with royalties paid to Authors. If there are other authors, they declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper].

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