Low-density lipoprotein receptor-deficient hepatocytes differentiated from induced pluripotent stem cells allow familial hypercholesterolemia modeling, CRISPR/Cas-mediated genetic correction, and productive hepatitis C virus infection.

Jérôme CaronVéronique PèneLaia TolosaMaxime VillaretEléanor LuceAngélique FourrierJean-Marie HeslanSamir SahebEric BruckertMaría José Gómez-LechónTuan Huy NguyenArielle R RosenbergAnne WeberAnne Dubart-Kupperschmitt

Published in: Stem cell research & therapy (2019)

Our work provides the first LDLR-null FH cell model and its corrected counterpart to study the regulation of cholesterol metabolism and host determinants of HCV life cycle, and a platform to screen drugs for treating dyslipidemia and HCV infection.

Keyphrases

low density lipoprotein
induced pluripotent stem cells
crispr cas
life cycle
hepatitis c virus infection
hepatitis c virus
high throughput
genome editing
single cell
cell therapy
liver injury
gene expression
drug induced
copy number
binding protein
dna methylation
wild type
hiv infected