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基于纳米聚合物的多功能向输送系统用于CRISPR/Cas9介导的肝细胞癌治疗

Gui Huang¹, Yan Tang², Shenghua Zhang³

¹Department of Pharmaceutics, College of Pharmaceutical Sciences, Soochow University, Jiangsu Suzhou 215123, China; Department of Pharmacy, The Affiliated Suzhou Hospital of Nanjing Medical University, Jiangsu Suzhou 215000, China.

International journal of pharmaceutics

|November 15, 2025

概括

一种新型的传递系统有效地利用CRISPR/Cas9基因编辑来向肝细胞癌 (HCC) 细胞,通过向METTL3.3来抑制瘤生长. 这种方法为HCC治疗提供了一个有前途的新策略.

科学领域:

生物技术是生物技术.
在瘤学瘤学.
基因治疗基因治疗

背景情况:

肝细胞癌 (HCC) 是一个重大的治疗挑战.
针对性地提供CRISPR/Cas9基因编辑系统用于HCC治疗至关重要.
甲基转移酶类3 (METTL3) 是HCC扩散的一个关键驱动因素.

研究的目的:

开发一种用于CRISPR/Cas9等离子体的多功能传递系统,以向HCC细胞中的METTL3.
提高基因编辑在HCC中的效率和特异性.
探索HCC治疗的新型治疗策略.

主要方法:

使用性PEI构建了一个输送系统,用脱氧胆酸 (DOCA) 修改为疏水性和乳酸 (LA) 修改为HCC细胞的积极向.
该系统通过对pH值敏感的聚乙烯糖醇 (PEG) 进一步功能化,以减少毒性和延长循环.
针对METTL3 (pMETTL3) 的CRISPR/Cas9等离子体被封装在输送系统 (pMETTL3/LPD) 中.

主要成果:

输送系统在生理pH下表现出稳定性,并增强了瘤组织中的积累.
观察到pMETTL3/LPD的有效细胞吸收,导致精确的基因编辑.
破坏METTL3表达导致诱导的亡和抑制HCC生长.

结论:

开发的多功能传递系统有效地准HCC细胞,并传递CRISPR/Cas9来破坏METTL3表达.
这种策略显示出抑制HCC生长的巨大潜力,并提供了一个有前途的治疗途径.
这项研究扩大了CRISPR/Cas9技术在癌症治疗中的应用.

关键词:

基因编辑CRISPR/Cas9基因编辑基因治疗是一种基因疗法.肝细胞癌是肝细胞癌.在METTL3中,METTL3是METTL3的第一个类型.纳米定向的交付提供.

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