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Updated: Jul 9, 2025

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

为了在大脑中进行基因组编辑,工程自发射的核糖核蛋白

Kai Chen, Elizabeth C Stahl, Min Hyung Kang

bioRxiv : the preprint server for biology

|November 28, 2023

概括

工程自发的CRISPR核糖核蛋白 (RNP) 提高了基因组编辑效率并降低了毒性. 这些新型RNP促进了在细胞中和在无辅助分子的情况下进行强大的基因编辑.

科学领域:

分子生物学分子生物学
基因编辑技术的技术
生物技术是生物技术.

背景情况:

克里斯普尔核糖核蛋白 (RNP) 为基因组编辑提供了优势,包括减少了目标外影响.
在特定细胞类型中,低效率和细胞毒性阻碍了有效的RNP传递.

研究的目的:

为增强细胞吸收和基因组编辑效率设计可自行交付的RNP.
开发一种传送系统,绕过对辅助材料的需求.

主要方法:

对与CRISPR-Cas9蛋白融合的细胞透 (CPPs) 的查.
工程的Cas9融合蛋白质,特别是C端融合与A22p的三个副本.
直接注射自发的Cas9 RNP到小鼠的条纹体,用于体内研究.

主要成果:

确定了强大的CPP-Cas9融合结构,用于神经前代细胞中高效的基因组编辑.
与三种A22p的Cas9融合显著提高了编辑效率.
自发的Cas9 RNPs在临床相关的基因中实现了强大的基因组编辑.

结论:

自发放的Cas9蛋白质代表了基因组编辑的一个简单有效的平台.
这种方法克服了RNP交付的挑战,使有效的体外和体内应用成为可能.

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Delivery of the Cas9/sgRNA Ribonucleoprotein Complex in Immortalized and Primary Cells via Virus-like Particles "Nanoblades"

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CRISPR

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...

RNA Editing

RNA Editing

RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...