Generation of a NONO homozygous knockout human induced pluripotent stem cell line by CRISPR/Cas9 editing

被引：2
|
作者：
Yi, Tong
Bai, Rui
Sun, Hairui
Lan, Feng
Zhang, Hongjia
He, Yihua
机构：
[1] Capital Med Univ, Beijing Anzhen Hosp, Beijing Key Lab Maternal Fetal Med & Fetal Heart, Beijing, Peoples R China
[2] Minist Educ, Key Lab Remodeling Related Cardiovasc Dis, Beijing Lab Cardiovasc Precis Med, Beijing, Peoples R China
[3] Capital Med Univ, Anzhen Hosp, Collaborat Innovat Ctr Cardiovasc Disorders, Beijing 100029, Peoples R China
来源：
STEM CELL RESEARCH | 2020年 / 47卷
基金：
国家重点研发计划;
关键词：
D O I：
10.1016/j.scr.2020.101893
中图分类号：
Q813 [细胞工程];
学科分类号：
摘要：
The non-POU domain containing octamer-binding gene (NONO) encodes a member of a small family of RNA-binding and DNA-binding proteins, whose variants can cause intellectual disability and congenital heart defects. In this study, we generated a homozygous NONO knockout (NONO-KO) induced pluripotent stem cell (iPSC) line (CMUi002-A-1) using the CRISPR/Cas9-based genome editing system. The gene-edited line had a normal karyotype, expressed pluripotency markers, and was able to differentiate into all three germ layers in vivo. This cell line will provide a platform to study the pathogenic mechanisms of noncompaction cardiomyopathy and neurocyte dysfunction related to NONO mutations.
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页数：4
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