The RNA revolution in medicine: from gene regulation to clinical therapeutics

Breakthrough discoveries in RNA biology have led to a paradigm shift in our understanding of RNA—from passive intermediates to active regulators of gene expression. Technological innovations and deeper insights into RNA regulation have transformed the field, positioning RNA as a powerful tool for th...

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Main Authors: Jiwon Jeong, Sunjoo Jeong
Format: Article
Language:English
Published: Taylor & Francis Group 2025-12-01
Series:Animal Cells and Systems
Subjects:
Online Access:https://www.tandfonline.com/doi/10.1080/19768354.2025.2548253
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author Jiwon Jeong
Sunjoo Jeong
author_facet Jiwon Jeong
Sunjoo Jeong
author_sort Jiwon Jeong
collection DOAJ
description Breakthrough discoveries in RNA biology have led to a paradigm shift in our understanding of RNA—from passive intermediates to active regulators of gene expression. Technological innovations and deeper insights into RNA regulation have transformed the field, positioning RNA as a powerful tool for therapeutic development. Recent advances in RNA technologies have revolutionized medicine by enabling the precise targeting of specific mRNAs to modulate aberrant transcripts, correct genetic defects, and reprogram cellular behavior. This review provides an overview of the coordinated regulation of mRNA processing and its application to RNA-based therapeutics, including antisense oligonucleotides (ASOs), splice-switching oligonucleotides (SSOs), small interfering RNAs (siRNAs), and Aptamers. We focus on clinically approved RNA therapeutics, emphasizing their biological mechanisms such as RNA stability and splicing regulation. The expanding repertoire of RNA technologies underscores the translational potential of RNA biology and its growing clinical impact. Future developments are expected to yield highly specific, modular, and programmable RNA medicines capable of treating a wide range of previously intractable diseases.
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spelling doaj-art-b2e614a13539483fba780b06d9a4d63a2025-08-25T07:58:57ZengTaylor & Francis GroupAnimal Cells and Systems1976-83542151-24852025-12-0129152354310.1080/19768354.2025.2548253The RNA revolution in medicine: from gene regulation to clinical therapeuticsJiwon Jeong0Sunjoo Jeong1Laboratory of RNA Cell Biology, Department of Bioconvergence Engineering, Dankook University, Yongin, Republic of KoreaLaboratory of RNA Cell Biology, Department of Bioconvergence Engineering, Dankook University, Yongin, Republic of KoreaBreakthrough discoveries in RNA biology have led to a paradigm shift in our understanding of RNA—from passive intermediates to active regulators of gene expression. Technological innovations and deeper insights into RNA regulation have transformed the field, positioning RNA as a powerful tool for therapeutic development. Recent advances in RNA technologies have revolutionized medicine by enabling the precise targeting of specific mRNAs to modulate aberrant transcripts, correct genetic defects, and reprogram cellular behavior. This review provides an overview of the coordinated regulation of mRNA processing and its application to RNA-based therapeutics, including antisense oligonucleotides (ASOs), splice-switching oligonucleotides (SSOs), small interfering RNAs (siRNAs), and Aptamers. We focus on clinically approved RNA therapeutics, emphasizing their biological mechanisms such as RNA stability and splicing regulation. The expanding repertoire of RNA technologies underscores the translational potential of RNA biology and its growing clinical impact. Future developments are expected to yield highly specific, modular, and programmable RNA medicines capable of treating a wide range of previously intractable diseases.https://www.tandfonline.com/doi/10.1080/19768354.2025.2548253RNAantisense oligonucleotidessplicing switching oligonucleotidesRNA interferenceaptamers
spellingShingle Jiwon Jeong
Sunjoo Jeong
The RNA revolution in medicine: from gene regulation to clinical therapeutics
Animal Cells and Systems
RNA
antisense oligonucleotides
splicing switching oligonucleotides
RNA interference
aptamers
title The RNA revolution in medicine: from gene regulation to clinical therapeutics
title_full The RNA revolution in medicine: from gene regulation to clinical therapeutics
title_fullStr The RNA revolution in medicine: from gene regulation to clinical therapeutics
title_full_unstemmed The RNA revolution in medicine: from gene regulation to clinical therapeutics
title_short The RNA revolution in medicine: from gene regulation to clinical therapeutics
title_sort rna revolution in medicine from gene regulation to clinical therapeutics
topic RNA
antisense oligonucleotides
splicing switching oligonucleotides
RNA interference
aptamers
url https://www.tandfonline.com/doi/10.1080/19768354.2025.2548253
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