Journal Article

RNA therapeutics: updates and future potential

Zhang, Caroline, Zhang, Biliang

2023

Overview

Recent advancements in the production, modification, and cellular delivery of RNA molecules facilitated the expansion of RNA-based therapeutics. The increasing understanding of RNA biology initiated a corresponding growth in RNA therapeutics. In this review, the general concepts of five classes of RNA-based therapeutics, including RNA interference-based therapies, antisense oligonucleotides, small activating RNA therapies, circular RNA therapies, and messenger RNA-based therapeutics, will be discussed. Moreover, we also provide an overview of RNA-based therapeutics that have already received regulatory approval or are currently being evaluated in clinical trials, along with challenges faced by these technologies. RNA-based drugs demonstrated positive clinical trial results and have the ability to address previously “undruggable” targets, which delivers great promise as a disruptive therapeutic technology to fulfill its full clinical potentiality.

• Publisher: Science China Press,Springer Nature B.V
• Subject: Antisense oligonucleotides / Antisense RNA / Antisense therapy / Biomedical and Life Sciences / Cellular manufacture / Chemistry / Circular RNA / Clinical trials / Cytomegalovirus / Disease prevention / Drugs / FDA approval / Life Sciences / Marketing / Medicine / MicroRNAs / mRNA / mRNA vaccines / Muscular dystrophy / Nobel prizes / Oligonucleotides / Oligonucleotides, Antisense - genetics / Oligonucleotides, Antisense - therapeutic use / Physiology / Proteins / Regulatory approval / Review / Ribonucleic acid / RNA / RNA Interference / RNA, Messenger - genetics / RNA, Small Interfering - genetics / RNA, Small Interfering - therapeutic use / RNA-mediated interference / Severe acute respiratory syndrome coronavirus 2 / Therapeutic applications