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脂质纳米粒子介导的CRISPR/Cas9传递能够在小鼠中高效地进行状状网状基因编辑

Yifan Huang¹, Linxian Li², Chi Wai Do³

¹Department of Biomedical Sciences, College of Biomedicine, City University of Hong Kong, Hong Kong, China.

Journal of controlled release : official journal of the Controlled Release Society

|December 4, 2025

概括

新的脂质纳米颗粒 (LNP) 有效地将基因编辑工具传递到眼睛的轨道眼网. 这种SM102-LNP平台在治疗青光眼和推进眼部基因治疗方面表现有前途.

科学领域:

眼部药物输送和基因疗法
纳米医学和生物材料
眼科和青光眼科的研究.

背景情况:

脂质纳米粒子 (LNP) 是已知的mRNA输送载体.
它们在眼部基因编辑中的应用尚未得到充分探索.
针对特定的眼组织,如状眼网 (TM) 是具有挑战性的.

研究的目的:

评估用于眼部基因传递的LNP配方.
评估基于SM102的LNP对TM向基因编辑的有效性和安全性.
开发一款用于初级开角绿眼病的临床前模型.

主要方法:

用mRNA对三个LNP配方 (DLin-MC3-DMA,ALC015,SM102) 的系统评估.
在眼细胞的体外转移和在小鼠体内的体内内注射.
在CRISPR-Cas9基因编辑中,将TM中的矩阵格拉蛋白 (Mgp) 淘汰.

主要成果:

基于SM102的LNP在眼细胞中表现出更高的转染效率.
静脉内SM102-LNP选择性地向TM,具有强大的,可重新诱导的表达.
比病毒载体,SM102-LNP显示出更好的TM特异性和较低的视网膜炎症.
通过SM102-LNPs引发的Mgp淘汰会引起类似于青光眼的特征,包括升高的IOP和视网膜应力.

结论:

基于SM102的LNP是一种安全有效的TM向基因传递平台.
这种LNP系统能够在眼睛中有效地进行CRISPR介导的基因编辑.
SM102-LNPs为眼模型和潜在的治疗策略提供了有价值的工具.

关键词:

基因编辑基因编辑静脉内注射静脉内注射脂质纳米粒子 (LNP) 是一种脊柱状的网状网状网状网状网状网.玻璃眼 glaucoma 玻璃眼玻璃眼玻璃眼玻璃眼它们是mRNARNA.

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