相关实验视频

Updated: Jan 13, 2026

CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation

CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation

Published on: June 20, 2019

FAST-CRISPR:使用脂质-混合纳米粒子用于治疗性基因组编辑的CRISPR/Cas9核核蛋白的融合性关联和安全传染

Minjong Kim¹, Kyunghwan Kim^2,3, Jihyun Lee⁴

¹Department of Biological Science, Ulsan National Institute of Science and Technology, Ulsan, Republic of Korea.

Small (Weinheim an der Bergstrasse, Germany)

|January 12, 2026

概括

一个新的FAST-CRISPR纳米粒子平台通过实现高效的细胞质递送和降低毒性来增强CRISPR/Cas9基因编辑. 这种非病毒方法对精确的基因组编辑疗法有希望.

科学领域:

生物技术是生物技术.
纳米医学是一种纳米医学.
基因编辑基因编辑

背景情况:

临床翻译CRISPR/Cas9基因编辑面临的挑战是,目前的病毒和纳米粒子载体的传递效率低下和毒性.
开发安全有效的非病毒传递系统对于推进CRISPR/Cas9疗法至关重要.

研究的目的:

开发和评估一种新的脂质-混合纳米粒子平台,即FAST-CRISPR,用于有效的CRISPR/Cas9核糖核蛋白的细胞质输送.
评估FAST-CRISPR纳米粒子在基因编辑,癌细胞亡诱导和瘤生长抑制方面的有效性和安全性.

主要方法:

使用DOTAP,DODMA脂质和大孔纳米颗粒制造脂质-混合纳米颗粒 (FAST-CRISPR).
评估纳米粒子融合性协会,等离子体膜融合,和细胞质递送绕过内细胞路径.
在体外和体内评估CRISPR/Cas9核核蛋白加载,分散,核运输和基因编辑效率.

主要成果:

快速CRISPR纳米粒子通过直接的血膜融合证明了优越的细胞内传递效率.
优化的脂质组成使得增强载荷,快速细胞溶液分散和Cas9/gRNA复合体的核运输成为可能.
多重基因组编辑诱导癌细胞亡,并显著抑制瘤生长在异种移植模型没有系统毒性.

结论:

快速CRISPR纳米粒子代表了一个安全和多功能的非病毒传递平台,用于精确的基因组编辑.
这项技术克服了CRISPR/Cas9治疗开发中的关键障碍,显示出显著的翻译潜力.

关键词:

这就是CRISPR/Cas9的作用.抗癌治疗药物抗癌治疗药物融合性的脂质体组组合体.有孔的二氧化纳米粒子.核蛋白是一种核蛋白.

更多相关视频

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

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy

Published on: March 12, 2018

相关概念视频

CRISPR/Cas9 Genome Editing

CRISPR/Cas9 Genome Editing

The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...

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...

Homologous Recombination

Homologous Recombination

The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...

CRISPR and crRNAs

CRISPR and crRNAs

Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...