相关实验视频

Updated: Jan 10, 2026

Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models

Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models

Published on: October 3, 2019

开发一种CRISPR/Cas9-degron系统,使得在白血病模型中能够在体内进行特定基因枯竭

Ruka Shimura¹, Keita Yamamoto¹, Yu-Hsuan Chang¹

¹Department of Computational Biology and Medical Sciences, Graduate School of Frontier Sciences, The University of Tokyo, Tokyo, Japan.

Biochemical and biophysical research communications

|November 22, 2025

概括

我们开发了一种新的Cas9-degron系统,用于精确控制CRISPR/Cas9基因组编辑的时间. 这个平台可以在急性髓性白血病模型中快速减少基因,突出SETDB1.

科学领域:

分子生物学分子生物学
遗传学是一个遗传学.
生物技术是生物技术.

背景情况:

克里斯普尔/卡斯9技术彻底改变了基因组编辑.
实现对Cas9活动的精确时间控制仍然是一个重大挑战.
现有的方法缺乏快速和可逆的Cas9失活的有效机制.

研究的目的:

为临时控制的CRISPR/Cas9基因组编辑开发一个Cas9-degron平台.
为了在急性髓性白血病 (AML) 模型中实现选择性体内基因枯竭.
在体内环境中调查SETDB1在AML进展中的作用.

主要方法:

开发一种Cas9降解系统,将降解标记的Cas9与dTAG化学降解策略相结合.
使用dTAG系统快速降解和随后恢复Cas9活动.
在AML模型 (MOLM13和cSAM) 中应用选择性体内基因枯竭系统.

主要成果:

在dTAG的存在下,Cas9-degron系统表现出快速且几乎完全的Cas9降解,在退出后恢复活性.
在AML模型中实现了选择性的体内基因枯竭.
证实了SETDB1,一个基因组H3K9甲基转移酶,对于体内AML细胞生长至关重要.
该系统通过控制移植后的SETDB1水平来避免培养诱导的预选择偏差.

结论:

Cas9-degron平台提供了对CRISPR/Cas9活动的精确时间控制,优于传统的Cas9系统.
这种多功能系统是体内CRISPR查和基因功能研究的强大工具.
该平台有可能开发临时控制的基因疗法.

更多相关视频

Investigation of Genetic Dependencies Using CRISPR-Cas9-based Competition Assays

Investigation of Genetic Dependencies Using CRISPR-Cas9-based Competition Assays

Published on: January 7, 2019

Electroporation-Based CRISPR-Cas9-Mediated Gene Knockout in THP-1 Cells and Single-Cell Clone Isolation

Electroporation-Based CRISPR-Cas9-Mediated Gene Knockout in THP-1 Cells and Single-Cell Clone Isolation

Published on: February 28, 2025

相关概念视频

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...

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...

In-vitro Mutagenesis

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.