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对于RNA输送的电离性脂质的合理设计和组合化学

Yue Xu¹, Alex Golubovic¹, Shufen Xu¹

¹Department of Pharmaceutical Sciences, Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada. bw.li@utoronto.ca.

Journal of materials chemistry. B

|June 22, 2023

概括

脂质纳米颗粒 (LNP) 对于提供RNA疗法至关重要. 本综述侧重于电离性脂质,mRNA疫苗和siRNA药物的LNP配方中的关键成分,探索它们的结构和合成.

科学领域:

生物技术是生物技术.
药物输送系统药物输送系统
核酸治疗药物核酸治疗药物

背景情况:

脂质纳米颗粒 (LNP) 已经彻底改变了核酸输送,使siRNA药物和mRNA疫苗的批准成为可能.
随着COVID-19的爆发,基于LNP的mRNA疫苗 (Comirnaty,Spikevax) 在公共卫生中发挥的关键作用得到了强调.
目前FDA批准的LNP配方除了可离子化脂质外,具有共同的结构成分.

研究的目的:

审查 RNA 输送的电离性脂质的最新进展.
探索电离性脂质的化学结构,合成途径和结构-活性关系.
为了强调可离子化脂质在基于LNP的治疗疗效中的重要性.

主要方法:

科学出版物和专利的文献评论.
分析电离性脂质的化学结构和合成路径.
在RNA输送的背景下检查结构-活性关系.

主要成果:

电离性脂质是成功的RNA输送LNP配方中的差异化成分.
在设计具有改进性质的新型电离性脂质方面取得了重大进展.
了解结构-活性关系是优化基于LNP的RNA疗法的关键.

结论:

电离性脂质是基于LNP的RNA传递效率的关键决定因素.
对电离性脂质设计的持续研究对于推进RNA疗法至关重要.
本综述提供了近几十年来RNA输送中电离性脂质的全面概述.

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