Elsevier

Stem Cell Research

Volume 55, August 2021, 102452
Stem Cell Research

Lab Resource: Genetically-Modified Single Cell Line
Generation of SCN1A Knock out induced pluripotent stem cell (iPSC) line

https://doi.org/10.1016/j.scr.2021.102452Get rights and content
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Abstract

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.

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