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细胞类型特定的线粒体DNA的CRISPR化,使用双功能可生物降解的二氧化纳米粒子

Linye Jiang¹, Bizhong Zhou¹, Huijuan Qian¹

¹Key Laboratory of Bioorganic Synthesis of Zhejiang Province, College of Biotechnology and Bioengineering, Zhejiang University of Technology, Hangzhou 310014, P. R. China. gejy@zjut.edu.cn.

Chemical communications (Cambridge, England)

|July 10, 2023

概括

这项研究引入了一个新的CRISPR/Cas9平台,用于精确的线粒体DNA编辑. 可生物降解的纳米粒子将编辑工具专门传递给目标细胞,从而实现有效的线粒体DNA修饰.

科学领域:

生物技术是生物技术.
分子生物学分子生物学
纳米医学是一种纳米医学.

背景情况:

线粒体DNA (mtDNA) 突变与各种疾病有关.
针对性和高效的mtDNA编辑仍然是一个挑战.
现有的传递系统缺乏细胞类型的特异性和线粒体向性.

研究的目的:

开发一种新的细胞类型特定和CRISPR/Cas9介导的mtDNA编辑平台.
为了在目标细胞内精确编辑线粒体DNA.
为了克服当前mtDNA编辑技术的局限性.

主要方法:

使用双功能可生物降解的纳米颗粒进行输送.
工程纳米粒子用于选择性细胞内输送到CD44过度表达的细胞.
实现了线粒体局部化和对Cas9/sgRNA释放的谷氨敏感生物降解.

主要成果:

证明了编辑系统的选择性传递到CD44过度表达的细胞.
在线粒体内成功定位了Cas9/sgRNA复合体.
通过释放的CRISPR/Cas9系统中介的精确mtDNA编辑得到确认.

结论:

开发的平台使细胞类型特定和线粒体向的mtDNA编辑成为可能.
可生物降解的纳米颗粒为线粒体中精确的基因操纵提供了一个有希望的方法.
这项技术在治疗线粒体疾病方面具有潜在的应用.

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