Department of Chemical Engineering, Pohang University of Science and Technology (POSTECH), 77 Cheongam-Ro, Nam-Gu, Pohang, Gyeongbuk, 37673, Korea.
Adv Healthc Mater. 2023 May;12(13):e2203033. doi: 10.1002/adhm.202203033. Epub 2023 Feb 20.
The recent development of RNA-based therapeutics in delivering nucleic acids for gene editing and regulating protein translation has led to the effective treatment of various diseases including cancer, inflammatory and genetic disorder, as well as infectious diseases. Among these, lipid nanoparticles (LNP) have emerged as a promising platform for RNA delivery and have shed light by resolving the inherent instability issues of naked RNA and thereby enhancing the therapeutic potency. These LNP consisting of ionizable lipid, helper lipid, cholesterol, and poly(ethylene glycol)-anchored lipid can stably enclose RNA and help them release into the cells' cytosol. Herein, the significant progress made in LNP research starting from the LNP constituents, formulation, and their diverse applications is summarized first. Moreover, the microfluidic methodologies which allow precise assembly of these newly developed constituents to achieve LNP with controllable composition and size, high encapsulation efficiency as well as scalable production are highlighted. Furthermore, a short discussion on current challenges as well as an outlook will be given on emerging approaches to resolving these issues.
近年来，基于 RNA 的治疗方法在递送用于基因编辑和调节蛋白质翻译的核酸方面取得了进展，为包括癌症、炎症和遗传疾病以及传染病在内的各种疾病的治疗带来了希望。在这些方法中，脂质纳米颗粒（LNP）已成为 RNA 递送的有前途的平台，并通过解决裸露 RNA 的固有不稳定性问题，从而提高治疗效果，为该领域带来了曙光。这些由可离子化脂质、辅助脂质、胆固醇和聚乙二醇锚定脂质组成的 LNP 可以稳定地包裹 RNA，并帮助它们释放到细胞的细胞质中。本文首先总结了从 LNP 成分、配方到其各种应用的 LNP 研究的重要进展。此外，还强调了微流控方法，这些方法可以精确组装这些新开发的成分，以实现具有可控组成和尺寸、高包封效率以及可扩展生产的 LNP。此外，还将对当前挑战以及解决这些问题的新兴方法进行简短的讨论。
