David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02142, USA; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02142, USA.
David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02142, USA; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02142, USA.
Med. 2022 Mar 11;3(3):167-187. doi: 10.1016/j.medj.2022.02.001.
RNA nanomedicines present a promising class of therapeutics, with broad applications in protein replacement therapy, gene editing, immunotherapy, and vaccines, owing to their versatility and precise nature. Although recent years have seen dramatic improvements in the safety and efficacy of RNA-based therapies, their functional delivery to target tissues and cells in vivo remains challenging. Here, we discuss many of these challenges, as well as the methods and materials that have been developed to overcome them, with a focus on polymeric and lipid-based nanoscale delivery systems. In addition, we provide an overview of recent clinical and pre-clinical developments in RNA nanomedicines that have been made possible by advances in delivery.
RNA 纳米药物是一类很有前途的治疗药物，由于其多功能性和精确性，在蛋白质替代疗法、基因编辑、免疫疗法和疫苗等方面有广泛的应用。尽管近年来 RNA 疗法的安全性和疗效有了显著提高，但在体内将其递送到靶组织和细胞仍然具有挑战性。在这里，我们讨论了其中的许多挑战，以及为克服这些挑战而开发的方法和材料，重点是聚合物和基于脂质的纳米级递药系统。此外，我们还概述了 RNA 纳米药物在临床前和临床方面的最新进展，这些进展得益于递药技术的进步。
