iPSC-derived hepatocytes accurately recapitulate population diversity in alpha-1-antitrypsin deficiency and offer a novel in vitro model for large-scale drug efficacy screening studies.
Carlos Gil, Vanesa Papastavrou, Gemma Gatti…·Nikolaos Nikolaou
This study generates iPSC-derived hepatocytes from A1ATD donors and shows they recapitulate disease features and respond to drug treatments, offering a model for drug screening.