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工程金属有机框架用于有针对性的CRISPR/Cas9基因编辑

Navid Rabiee^1,2,3,4, Mohammad Rabiee⁵

¹Department of Basic Medical Science, School of Medicine, Tsinghua University, Beijing 100084, China.

ACS pharmacology & translational science

|April 17, 2025

概括

工程金属有机框架 (MOF) 为CRISPR/Cas9基因编辑提供了先进的交付,提高了精度和效率. 这些生物相容的纳米平台显示出在精密医学中更安全,更有针对性的基因治疗的前景.

科学领域:

纳米技术纳米技术
生物医学工程生物医学工程
分子生物学分子生物学

背景情况:

对于治疗用途,CRISPR/Cas9基因编辑需要高效和精确的输送系统.
非病毒载体对于安全有效的基因传递至关重要,但往往面临局限性.
金属有机框架 (MOFs) 是一种具有可调节性质的新型无机纳米载体.

研究的目的:

审查用于CRISPR/Cas9交付的基于MOF的纳米平台的设计和应用.
突出MOF在提高基因编辑精度和效率方面的潜力.
讨论基于MOF的系统作为先进的非病毒治疗输送载体.

主要方法:

探索MOF设计策略,包括刺激响应链接器和生物活性连接物.
对基于MOF的纳米平台进行分析,以有针对性地提供CRISPR/Cas9组件.
对评估MOF性能与传统非病毒系统相比的比较研究的综述.
讨论细胞内贩运和临床前治疗潜力.

主要成果:

MOFs表现出可调节的多孔性,高载荷能力和良好的生物相容性.
基于MOF的系统可以在目标地点控制释放CRISPR/Cas9有效载荷.
与传统载体相比,MOFs表现出优越的稳定性,转染效率和减少的目标外影响.
MOF输送系统显示出克服免疫性和细胞毒性问题的潜力.

结论:

基于MOF的纳米平台代表了CRISPR/Cas9交付的变革性方法.
这些系统为基因编辑疗法提供了更高的精度,效率和安全性.
MOFs为精准医学中先进的非病毒治疗输送系统铺平了道路.

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