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一个基于DNA原始的基因编辑系统,用于有效的基因疗法 in vivo

Wantao Tang^1,2, Ting Tong², Hong Wang²

¹School of Materials Science and Engineering, Zhengzhou University, Zhengzhou, 450001, China.

Angewandte Chemie (International ed. in English)

|October 31, 2023

概括

这项研究引入了用于CRISPR/Cas9基因编辑的DNA原始体系统,使得向基因疗法 in vivo. 纳米结构有效地提供基因编辑工具,并在瘤细胞中实现显著的基因修饰.

科学领域:

生物技术是生物技术.
纳米技术纳米技术
分子生物学分子生物学

背景情况:

DNA纳米结构对于先进的药物输送系统至关重要.
克里斯普尔/卡斯9技术提供了强大的基因编辑能力.
有效和有针对性的基因编辑工具的交付仍然是一个挑战.

研究的目的:

开发一种基于DNA原形的CRISPR/Cas9基因编辑系统,用于有效的体内基因治疗.
为精确招募,加载和控制释放CRISPR/Cas9复合物的纳米结构进行工程.
为了实现有针对性的分娩和增强内体逃生,以改善治疗结果.

主要方法:

制造具有PAM丰富表面的DNA原始结构纳米结构,用于sgRNA/Cas9复合体的招募.
通过PAM引导组装和DNA/RNA杂交,加载sgRNA/Cas9复合体.
用于卷起纳米结构的二硫化物键形成,其次是以阿普坦和HA为目标输送的合并.
通过GSH和RNase H分裂触发的sgRNA/Cas9复合物的受控释放,用于基因编辑.

主要成果:

成功组装并将sgRNA/Cas9复合体加载到DNA原始体上.
向的输送和高效的内分体逃逸,由阿普坦和HA促进.
由GSH诱导的开放和RNase H分裂导致了基因编辑复合物的释放.
在体内证明了瘤相关基因的明显基因编辑,证实了治疗潜力.

结论:

开发的DNA原形系统为CRISPR/Cas9基因编辑 in vivo提供了一个高效的平台.
这种方法为向基因治疗提供了一种新的策略,具有增强的传递和释放机制.
该系统为推进基因治疗应用提供了一个有希望的新途径.

关键词:

基因原始是指DNA的原始形式.药物运输药物运输药物运输基因编辑基因编辑基因治疗基因治疗自动组装自动组装

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