RNA-based therapies

RNA-based therapeutics have generated significant attention in recent years due to their potential to treat a variety of chronic and rare diseases, and address targets that have proven to be intractable to antibody and small-molecule approaches. To highlight advances in delivery technologies and improved chemistry that have propelled the clinical advancement of RNA-based therapies (antisense, siRNAs, aptamers, microRNA mimics/anti-miRs and synthetic mRNA), Nature Biotechnology and Nature Reviews Drug Discovery present a joint focus on RNA-based therapies.