从脂质纳米粒子的角度看 RNA 的传递

Exploration Pub Date : 2024-04-15 DOI:10.1002/exp.20230147
Yutian Ma, Shiyao Li, Xin Lin, Yupeng Chen
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引用次数: 0

摘要

在过去的二十年里,脂质纳米颗粒(LNPs)已发展成为纳米医学、生物技术和药物输送领域中一种有效的生物相容性和可生物降解的 RNA 输送平台。LNPs 是一种新型仿生材料,可用于封装多种生物大分子,如 mRNA,目前 COVID-19 mRNA 疫苗的成功就证明了这一点。因此,有必要对用于递送 RNA 的 LNPs 进行透视,从而为希望在基于 RNA 的 LNP 领域开展工作的研究人员提供有用的指导。本视角首先总结了 LNPs 的制备方法,然后介绍了关键的表征参数。然后,总结了研究 LNP 性能的体外细胞实验,包括细胞选择、细胞活力、细胞关联/摄取、内体逸出及其功效。最后,从动物选择、给药、剂量和安全性及其疗效等方面讨论了体内动物实验。作者希望这一观点能为进入基于 RNA 的 LNPs 领域的研究人员提供有价值的指导,帮助他们了解基于 RNA 的 LNPs 所需的关键参数。
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A perspective of lipid nanoparticles for RNA delivery
Over the last two decades, lipid nanoparticles (LNPs) have evolved as an effective biocompatible and biodegradable RNA delivery platform in the fields of nanomedicine, biotechnology, and drug delivery. They are novel bionanomaterials that can be used to encapsulate a wide range of biomolecules, such as mRNA, as demonstrated by the current successes of COVID‐19 mRNA vaccines. Therefore, it is important to provide a perspective on LNPs for RNA delivery, which further offers useful guidance for researchers who want to work in the RNA‐based LNP field. This perspective first summarizes the approaches for the preparation of LNPs, followed by the introduction of the key characterization parameters. Then, the in vitro cell experiments to study LNP performance, including cell selection, cell viability, cellular association/uptake, endosomal escape, and their efficacy, were summarized. Finally, the in vivo animal experiments in the aspects of animal selection, administration, dosing and safety, and their therapeutic efficacy were discussed. The authors hope this perspective can offer valuable guidance to researchers who enter the field of RNA‐based LNPs and help them understand the crucial parameters that RNA‐based LNPs demand.
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