Nanomaterials for Therapeutic RNA Delivery

Abstract

Recent years have witnessed tremendous developments and breakthroughs in the field of RNA-based therapeutics The distinct mechanisms of exogenous RNAs and analogs, including messenger RNAs, small interfering RNAs, microRNAs, and antisense oligonucleotides, have brought them unprecedented potential to treat a variety of pathological conditions However, the widespread application of RNA therapeutics is hampered by their intrinsic features (e g , instability, large size, and dense negative charge) and formidable host barriers Development of safe and efficient vectors is key for successful delivery and translation of RNA therapeutics In this review, we first present an overview of RNA therapeutics and their clinical translation We then discuss their delivery challenges and highlight recent advances in nanomaterial-based RNA-delivery platforms Finally, the potential concerns and future developments of RNA delivery systems are discussed © 2020 Elsevier Inc RNA therapeutics hold great promise to treat many diseases including infectious diseases, cancers, and genetic disorders However, their intrinsic properties (e g , instability and large size) and formidable barriers have hindered their broad applications There is a growing demand for developing safe and efficient vectors for RNA delivery Nanomaterials (NMs), with the potential to overcome multiple physiological barriers, have emerged as promising non-viral vectors With one marketed RNA drug, patisiran, and many drug candidates in clinical trials, NM-based delivery platforms are bridging the gap between RNA molecules and medicinal applications Specifically, the unprecedented speed of mRNA vaccine development against the COVID-19 pandemic addresses the great value of NMs In this review, we first give an overview of RNA therapeutics and their clinical translation We then summarize recent advances in RNA-delivery NMs Finally, we provide perspectives on future developments of NMs © 2020 Elsevier Inc Recent years have witnessed tremendous development and breakthroughs in the field of RNA-based therapeutics The distinct mechanisms of exogenous RNAs and analogs, including messenger RNAs, small interfering RNAs, microRNAs, and antisense oligonucleotides, have brought them unprecedented potential to treat a variety of pathological conditions However, the widespread application of RNA therapeutics is hampered by their intrinsic features (e g , instability, large size, and dense negative charge) and formidable host barriers Development of safe and efficient vectors is key for successful delivery and translation of RNA therapeutics In this review, we first present an overview of RNA therapeutics and their clinical translation We then discuss their delivery challenges and highlight recent advances in nanomaterial-based RNA-delivery platforms Finally, the potential concerns and future developments of RNA-delivery systems are discussed © 2020 Elsevier Inc