RNA-based therapeutics: cutting-edge advances in clinical research

Abstract

Over the years, the relevance of gene therapy has increased as an alternative to conventional drugs. Pharmaceutical industries have been directing their efforts toward the creation of novel gene-based therapies for a broad range of diseases. RNA-based therapeutics can efficiently trigger gene silencing and, despite the initial research setbacks, this technology laid the groundwork for advancements in RNA-based drug design that ultimately contributed to the remarkably rapid advance of messenger RNA (mRNA) vaccines to combat the COVID-19 pandemic. In light of this information, RNA medicines are currently well-positioned to have a noteworthy impact in clinical practice. Various RNA medicines have received clinical approval, while others are still in the research phase or undergoing preclinical trials. In this review, an overview of RNA-based therapeutics is aimed to provide, including antisense oligonucleotides and RNA interference-based mechanisms, mRNA-encoded approaches, aptamers, and clustered regularly interspaced short palindromic repeats (CRISPR-Cas)-mediated genome editing. Furthermore, the commonly used chemistries in drug delivery systems and strategies for targeting specific cells, as well as the key nanocarriers used in RNA loading, are explored. The main advances and current challenges will be thoroughly summarized.