Wu Shiqi, Shi Lu, Su Kexin, Lin Lixin, Yan Xinxin, Zhang Yiran, Gu Tianrui, Wang Zichuan, Xu Tengfei, Liu Shuai
Eye Center of the Second Affiliated Hospital of Zhejiang University School of Medicine, College of Pharmaceutical Sciences, Liangzhu Laboratory, Zhejiang University, Hangzhou 310058, China.
State Key Laboratory of Advanced Drug Delivery and Release Systems, Zhejiang University, Hangzhou 310058, China.
J Am Chem Soc. 2025 Aug 13;147(32):28665-28673. doi: 10.1021/jacs.5c01389. Epub 2025 Jun 11.
mRNA immunotherapies targeting natural killer (NK) cells hold substantial potential for treating cancer and viral diseases. However, achieving functional delivery of mRNA to splenic NK cell subsets remains a formidable challenge. Herein, we rationally design a library of 161 carbonate-bearing ionizable lipids (CAILs) and formulate lipid nanoparticles (LNPs) for functional mRNA delivery to the spleen and NK cells following intravenous administration. Unlike traditional LNPs that primarily target the liver, CAIL-based four-component LNPs predominantly deliver mRNA to the spleen. Notably, the removal of cholesterol from CAIL LNPs further enhances the spleen-targeting specificity and efficacy, significantly surpassing those of previous spleen-targeted LNPs. As a result, the optimized CAIL LNPs achieve robust mRNA translation in splenic NK cells, inducing encouraging transfection of 21% of splenic NK cells in an Ai9 mouse model. The lipid structural design and LNP formulation optimization pave the way to address the challenge of efficient mRNA delivery to NK cells, presenting opportunities for the development of NK cell-based mRNA therapies.
靶向自然杀伤(NK)细胞的信使核糖核酸(mRNA)免疫疗法在治疗癌症和病毒性疾病方面具有巨大潜力。然而,实现mRNA向脾NK细胞亚群的功能性递送仍然是一项艰巨的挑战。在此,我们合理设计了一个包含161种含碳酸根的可电离脂质(CAIL)的文库,并制备了脂质纳米颗粒(LNP),用于在静脉注射后将功能性mRNA递送至脾脏和NK细胞。与主要靶向肝脏的传统LNP不同,基于CAIL的四组分LNP主要将mRNA递送至脾脏。值得注意的是,从CAIL LNP中去除胆固醇进一步提高了脾脏靶向特异性和疗效,显著超过了先前的脾脏靶向LNP。结果,优化后的CAIL LNP在脾NK细胞中实现了强大的mRNA翻译,在Ai9小鼠模型中诱导了21%的脾NK细胞实现令人鼓舞的转染。脂质结构设计和LNP制剂优化为解决向NK细胞高效递送mRNA的挑战铺平了道路,为基于NK细胞的mRNA疗法的发展提供了机遇。