Center for RNA Nanobiotechnology and Nanomedicine, The Ohio State University, Columbus, Ohio.
Division of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, Ohio.
Wiley Interdiscip Rev Nanomed Nanobiotechnol. 2020 Jan;12(1):e1582. doi: 10.1002/wnan.1582. Epub 2019 Aug 27.
The past decade has shown exponential growth in the field of RNA nanotechnology. The rapid advances of using RNA nanoparticles for biomedical applications, especially targeted cancer therapy, suggest its potential as a new generation of drug. After the first milestone of small molecule drugs and the second milestone of antibody drugs, it was predicted that RNA drugs, either RNA itself or chemicals/ligands that target RNA, will be the third milestone in drug development. Thus, a comprehensive assessment of the current therapeutic RNA nanoparticles is urgently needed to meet the drug evaluation criteria. Specifically, the pharmacological and immunological profiles of RNA nanoparticles need to be systematically studied to provide insights in rational design of RNA-based therapeutics. By virtue of its programmability and biocompatibility, RNA molecules can be designed to construct sophisticated nanoparticles with versatile functions/applications and highly tunable physicochemical properties. This intrinsic characteristic allows the systemic study of the effects of various properties of RNA nanoparticles on their in vivo behaviors such as cancer targeting and immune responses. This review will focus on the recent progress of RNA nanoparticles in cancer targeting, and summarize the effects of common physicochemical properties such as size and shape on the RNA nanoparticles' biodistribution and immunostimulation profiles. This article is categorized under: Biology-Inspired Nanomaterials > Nucleic Acid-Based Structures Diagnostic Tools > in vivo Nanodiagnostics and Imaging Therapeutic Approaches and Drug Discovery > Nanomedicine for Oncologic Disease.
过去十年，RNA 纳米技术领域呈现出指数级的增长。RNA 纳米颗粒在生物医学应用，特别是靶向癌症治疗方面的快速发展，表明其具有成为新一代药物的潜力。继小分子药物的第一个里程碑和抗体药物的第二个里程碑之后，有人预测，RNA 药物，无论是 RNA 本身还是靶向 RNA 的化学物质/配体，都将成为药物开发的第三个里程碑。因此，迫切需要对当前的治疗性 RNA 纳米颗粒进行全面评估，以满足药物评价标准。具体来说，需要系统研究 RNA 纳米颗粒的药理学和免疫学特性，为基于 RNA 的治疗剂的合理设计提供深入了解。由于其可编程性和生物相容性，RNA 分子可以被设计用来构建具有多种功能/应用和高度可调理化性质的复杂纳米颗粒。这种内在特性允许系统地研究 RNA 纳米颗粒的各种性质对其体内行为（如癌症靶向和免疫反应）的影响。本文将重点介绍 RNA 纳米颗粒在癌症靶向方面的最新进展，并总结常见理化性质（如大小和形状）对 RNA 纳米颗粒的生物分布和免疫刺激特性的影响。本文属于以下分类：生物启发型纳米材料 > 基于核酸的结构 诊断工具 > 体内纳米诊断和成像 治疗方法和药物发现 > 用于肿瘤疾病的纳米医学。
