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Updated: Jan 9, 2026

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核心外三脂/PEI纳米载体使基于等离子体的CRISPR/Cas9能够有效编辑VEGFR2

Zhao Gao¹, Kexin Lin¹, Yan Gong¹

¹Key Lab of Biotechnology and Bioresources Utilization of Ministry of Education, College of Life Science, Dalian Minzu University, Dalian, Liaoning 116600, China.

Langmuir : the ACS journal of surfaces and colloids

|December 10, 2025

概括

一个新的三元复合系统有效地使用CDO和PEI提供CRISPR/Cas9基因编辑组件,显示出高转染效率和减少潜在癌症治疗的细胞毒性.

科学领域:

生物技术和基因工程生物技术和基因工程
纳米医学和药物输送系统
分子生物学和癌症研究分子生物学和癌症研究

背景情况:

CRISPR/Cas9是一种强大的基因组编辑工具,对遗传疾病具有治疗潜力.
对CRISPR/Cas9组件的无效体内传递仍然是临床应用的重大挑战.
现有的传递方法,如病毒载体和脂质纳米颗粒,在效率和安全方面存在局限性.

研究的目的:

开发和评估用于增强CRISPR/Cas9交付的新型三元系统.
研究这些系统在提供向VEGFR2基因的CRISPR/Cas9组件方面的有效性.
在癌症模型中评估这种交付策略的治疗潜力.

主要方法:

使用三脂CDO和聚乙烯胺 (PEI) 构建三元系统 (pDNA/PEI/CL).
用PEI对编码Cas9和sgRNA的pDNA进行凝聚,然后用基于CDO的阴阳性脂质体进行涂层.
在细胞系和动物模型中评估转染效率,细胞毒性,基因组编辑效率和抗瘤效应.

主要成果:

该pDNA/PEI/CL系统在低细胞毒性A549和MCF-7细胞中实现了高传染效率 (高达91.0%).
特定的sgRNA复合体显示出显著的基因组编辑效率,导致VEGFR2和PI3K表达的减少.
抑制PI3K/Akt通路导致细胞循环停止,抑制瘤细胞的增殖和迁移,并促进细胞亡,在体内得到验证的抗瘤疗效.

结论:

开发的三元复杂系统是CRISPR/Cas9传递的强大和生物相容的策略.
这个平台显示了在癌症治疗中基因疗法应用的巨大潜力.
这些发现为推进各种遗传疾病的CRISPR/Cas9疗法铺平了道路.

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