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Lipid nanoparticles for mRNA delivery
Nature Reviews Materials ( IF 83.5 ) Pub Date : 2021-08-10 , DOI: 10.1038/s41578-021-00358-0
Xucheng Hou 1 , Tal Zaks 2 , Robert Langer 3, 4 , Yizhou Dong 1
Affiliation  

Messenger RNA (mRNA) has emerged as a new category of therapeutic agent to prevent and treat various diseases. To function in vivo, mRNA requires safe, effective and stable delivery systems that protect the nucleic acid from degradation and that allow cellular uptake and mRNA release. Lipid nanoparticles have successfully entered the clinic for the delivery of mRNA; in particular, lipid nanoparticle–mRNA vaccines are now in clinical use against coronavirus disease 2019 (COVID-19), which marks a milestone for mRNA therapeutics. In this Review, we discuss the design of lipid nanoparticles for mRNA delivery and examine physiological barriers and possible administration routes for lipid nanoparticle–mRNA systems. We then consider key points for the clinical translation of lipid nanoparticle–mRNA formulations, including good manufacturing practice, stability, storage and safety, and highlight preclinical and clinical studies of lipid nanoparticle–mRNA therapeutics for infectious diseases, cancer and genetic disorders. Finally, we give an outlook to future possibilities and remaining challenges for this promising technology.



中文翻译:

用于 mRNA 递送的脂质纳米粒子

信使RNA(mRNA)已成为预防和治疗各种疾病的一类新的治疗剂。为了在体内发挥作用,mRNA 需要安全、有效和稳定的递送系统,以保护核酸免遭降解,并允许细胞摄取和 mRNA 释放。脂质纳米粒子已成功进入临床用于mRNA的递送;特别是,脂质纳米颗粒-mRNA疫苗现已投入临床用于对抗2019年冠状病毒病(COVID-19),这标志着mRNA疗法的一个里程碑。在这篇综述中,我们讨论了用于 mRNA 递送的脂质纳米颗粒的设计,并检查了脂质纳米颗粒-mRNA 系统的生理障碍和可能的给药途径。然后,我们考虑脂质纳米颗粒-mRNA制剂临床转化的关键点,包括良好的生产规范、稳定性、储存和安全性,并重点介绍脂质纳米颗粒-mRNA疗法用于传染病、癌症和遗传性疾病的临床前和临床研究。最后,我们展望了这项有前途的技术的未来可能性和仍然面临的挑战。

更新日期:2021-08-10
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