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深圳星盛生物科技有限公司

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№ CN122937027AInvention
Unknown

一种脑细胞特异性mRNA及CRISPR脂质纳米颗粒及其协同逆向设计方法与系统

本发明提供了一种脑细胞特异性mRNA及CRISPR脂质纳米颗粒及其协同逆向设计方法与系统，所述脂质纳米颗粒包括脂质纳米颗粒核心、包封于核心中的核酸载荷及设置于颗粒表面的多配体组件；用于脑微血管内皮捕获的第一血脑屏障配体、用于调控内吞分选路径的第二协同转胞吞配体、以及经环境响应屏蔽层遮蔽的脑细胞特异性配体，本发明构建耦合血流剪切、蛋白冠动力学、跨胞运输及胞内效应的脑微血管数字孪生体，结合生成式肽设计模型与多保真主动学习，实现脂质组成、颗粒参数、配体组合及核酸载荷结构的联合优化。本发明显著提高了核酸载荷跨越血脑屏障的效率及脑细胞选择性，降低了外周脱靶效应与内皮滞留，适用于脑部疾病的精准基因治疗与研究试剂开发。

❖深圳星盛生物科技有限公司魏玮琛; 刘婕妤Oct 20, 2026§ 4/10 ClaimsA61K 9/51 (2006.01), A61K 48/00 (2006.01), A61K 31/7105 (2006.01) +13