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生物模拟矿物化CRISPR/CasRNA纳米粒子,用于高效的瘤特异多重复基因编辑

Yan Liang¹, Jingge Zhang¹, Chenlu Xu¹

¹School of Pharmaceutical Sciences, Key Laboratory of Targeting Therapy and Diagnosis for Critical Diseases Collaborative Innovation Center of New Drug Research and Safety Evaluation, Zhengzhou University, Zhengzhou 450001, P. R. China.

|July 23, 2023

概括

研究人员开发了一种用于CRISPR/Cas9RNA传递的新型生物模拟矿物化系统,可在体内进行高效的多重基因编辑. 该系统精确地提供Cas9mRNA和多个导向RNA,增强复杂遗传疾病的治疗潜力.

科学领域:

生物材料科学生物材料科学
基因治疗基因治疗
纳米技术纳米技术

背景情况:

CRISPR/Cas9基因编辑需要有效地传递多个RNA组件.
现有的交付系统面临着在多重编辑时的石化度,容量和稳定性方面的挑战.
生物矿物化为创造先进生物材料提供了灵感.

研究的目的:

为CRISPR/Cas9RNA传递开发一个全合一的仿生矿物化系统.
为了实现Cas9mRNA和多个sgRNA的高效协同传递,用于多重基因编辑.
证明开发的输送系统的 in vivo 疗效和安全性.

主要方法:

由自然过程启发的生物仿真矿化.
同时封装Cas9mRNA和多个sgRNA在受控的比例下.
纳米颗粒的表面功能化用于有针对性的交付.
在小鼠模型中的体内测试用于基因编辑和瘤抑制.

主要成果:

高RNA负载能力和精确的联合封装比率控制.
在4°C下提高RNA稳定性,持续一个月.
在HeLa细胞中的目标位点 (Survivin,PLK1,HPV) 实现了显著的基因编辑.
在体内抑制瘤生长,在肝脏组织中产生最小的非目标效应.

结论:

开发的生物模拟矿物化系统有效地解决了CRISPR/Cas9.9的多元组件RNA传递方面的挑战.
这一创新策略使得有效的体内多重基因编辑和治疗应用成为可能.
该系统显示了先进的基因治疗和细胞工程的前景.

关键词:

在Cas9 mRNA输送过程中,mRNA 在RNA纳米技术上.RNA-无机杂交复合材料的组合物生物材料是一种生物材料.生物模拟矿物化的生物模拟矿物化提高mRNA的稳定性,提高mRNA的稳定性.多重基因编辑多重基因编辑

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