Generation of an iPSC-line (BIONi010C-48) with restored P-glycoprotein functionality following transfection with the human MDR1 gene in the AAVS1 locus

Amanda Kongstad Redke Nielsen, Laura Lilieholm-Rongren,Benjamin Schmid,Bjorn Holst, Birger Brodin,Lasse Saaby

STEM CELL RESEARCH(2024)

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摘要
The human MDR1 gene encodes the efflux transporter P-glycoprotein, which plays an important part of the blood-brain barrier function of brain microvascular endothelial cells (BMECs). Here, we report the generation of an iPSC line, where a construct of the human MDR1 gene was inserted into the safe -site locus AAVS1. This iPSC line (BIONi010-C-48) shows functional expression of P-gp and can further be differentiated and cultured into electrically tight BMEC-like monolayers exhibiting polarized expression of P-gp in the apical membrane.
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