相关实验视频

Updated: Jun 29, 2026

Efficient Generation of hiPSC Neural Lineage Specific Knockin Reporters Using the CRISPR/Cas9 and Cas9 Double Nickase System

Efficient Generation of hiPSC Neural Lineage Specific Knockin Reporters Using the CRISPR/Cas9 and Cas9 Double Nickase System

Published on: May 28, 2015

使用CRISPR/Cas9系统建立一个同胞性LMNA淘汰人类诱导的多能干细胞系

So Hee Park¹, David Suh², Hyoeun Kim¹

¹Department of Biochemistry and Molecular Biology, Yonsei University College of Medicine, 50-1 Yonsei-ro, Seodaemun-gu, Seoul 03722, South Korea.

Stem cell research

|July 24, 2025

概括

研究人员创造了一个人类诱导的多能干细胞系,在LMNA基因中发生了突变. 这种LMNA淘汰模型有助于研究核包障碍及其对细胞健康的影响.

科学领域:

细胞生物学细胞生物学
遗传学遗传学是一个
生物化学生物化学

背景情况:

这种LMNA基因提供了制造称为lamin A和C的蛋白质的指令.
这些蛋白质是核外的重要组成部分,对核结构,细胞信号和基因表达至关重要.
在LMNA的突变导致拉米诺病,一组影响多个器官的遗传性疾病.

研究的目的:

开发一种人类细胞模型,用于研究LMNA相关疾病.
为了研究层A/C在核完整性和细胞平衡中的功能.

主要方法:

使用CRISPR/Cas9基因组编辑,将一个框架转移突变 (c.351_352insA) 引入LMNA基因.
一个人类诱导的多能干细胞 (hiPSC) 线因同卵性LMNA突变而产生.
编辑的hiPSCs的特征是多能性标记物和殖民地形态.

主要成果:

一种同卵性LMNA框架转移突变成功地引入了hiPSCs.
由此产生的LMNA淘汰赛hiPSC线保持正常的形态和表达关键的多能性标志物.
这种工程细胞系作为LMNA相关疾病的模型.

结论:

生成的LMNA淘汰赛hiPSC线是研究层A/C的作用的宝贵工具.
这种模型将有助于研究层状病变的发病因子.
进一步的研究可以利用这种模型探索核完整性,细胞平衡和治疗策略.

更多相关视频

Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells

Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells

Published on: September 25, 2019

CRISPR-Cas9 Mediated Gene Deletion in Human Pluripotent Stem Cells Cultured Under Feeder-Free Conditions

CRISPR-Cas9 Mediated Gene Deletion in Human Pluripotent Stem Cells Cultured Under Feeder-Free Conditions

Published on: November 1, 2024

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 Short...

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...