A Drug Screen using Human iPSC-Derived Hepatocyte-like Cells Reveals Cardiac Glycosides as a Potential Treatment for Hypercholesterolemia

Max A. Cayo, Sunil K. Mallanna, Francesca Di Furio, Ran Jing, Lauren B. Tolliver, Matthew Bures, Amanda Urick, Fallon K. Noto, Evanthia E. Pashos, Matthew D. Greseth, Maciej Czarnecki, Paula Traktman, Wenli Yang, Edward E. Morrisey, Markus Grompe, Daniel J. Rader, Stephen A. Duncan

Index: 10.1016/j.stem.2017.01.011

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Abstract

Cayo et al. of the NHLBI NextGen consortium use hepatocytes from familial hypercholesterolemia iPSCs in a drug screen that highlights cardiac glycosides as a candidate treatment acting to reduce apoB levels via proteolytic turnover and as a result lower LDL-C.