Generation of SCN1A Knock out induced pluripotent stem cell (iPSC) line

被引:4
|
作者
Shan, Wei [1 ,2 ]
Yang, Xiaoling [3 ]
Ren, Qian [4 ,5 ,6 ]
Wang, Qun [1 ,2 ]
机构
[1] Capital Med Univ, Dept Neurol, Beijing Tiantan Hosp, Beijing 100070, Peoples R China
[2] Natl Ctr Clin Med Neurol Dis, Beijing 10070, Peoples R China
[3] Peking Univ First Hosp, Dept Pediat, 1 Xian Men St, Beijing 100034, Peoples R China
[4] Hebei Med Univ, Dept Human Anat, Shijiazhuang 050017, Hebei, Peoples R China
[5] Hebei Med Univ, Inst Med & Hlth Sci, Int Cooperat Lab Stem Cell Res, Shijiazhuang 050017, Hebei, Peoples R China
[6] Res Ctr Stem Cell Med Translat Engn, Shijiazhuang 050017, Hebei, Peoples R China
基金
中国博士后科学基金; 中国国家自然科学基金;
关键词
D O I
10.1016/j.scr.2021.102452
中图分类号
Q813 [细胞工程];
学科分类号
摘要
The SCN1A gene encodes the voltage-gated Na+ channel alpha subunit Nav1.1 and is the most clinically relevant epilepsy gene. Variants in SCN1A result in a broad phenotypic spectrum of epilepsy syndromes, from mild genetic epilepsy with febrile seizures plus to severe Dravet syndrome (DS). Here, we generated a SCN1A-knockout human iPSC line via CRISPR/Cas9 gene editing. The resulting iPSCs had a normal karyotype, were free of genomically integrated epitomal plasmids, expressed pluripotency markers, and maintained trilineage differentiation potential.
引用
收藏
页数:4
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