Journal of Biomolecular Research & Therapeutics

Abstract

Advances in RNA Therapeutics and Their Clinical Applications

Sophie Dupont

RNA-based therapeutics have emerged as an important class of treatments, offering unique advantages over traditional drugs. Unlike small molecules or protein therapies, RNA therapies work by directly modulating gene expression, allowing precise targeting of disease mechanisms at the molecular level. The ability to influence gene activity opens up exciting possibilities for treating a wide range of conditions, particularly those caused by genetic mutations, where traditional therapies often fall short. One of the earliest RNA-based therapies was antisense oligonucleotides. These short strands of synthetic nucleotides bind to messenger RNA, preventing the production of disease causing proteins. Approved therapies now exist for conditions such as spinal muscular atrophy and Duchenne muscular dystrophy, demonstrating the clinical potential of this approach. These therapies offer patients with rare genetic diseases the opportunity for life-changing treatments, where previously there were no viable options

Published Date: 2025-06-28; Received Date: 2025-05-29