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

Abstract

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.