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. 2022 Sep 8;66(1):12–30. doi: 10.1007/s11427-022-2171-2

RNA therapeutics: updates and future potential

Caroline Zhang 2, Biliang Zhang 1,3,✉
PMCID: PMC9470505  PMID: 36100838

Abstract

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.

Keywords: RNA, RNA therapeutics, mRNA vaccine, RNAi

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