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, Samuel Chung, George Kiloh, Ka Chun Cheung, Magdalena Łukasiak, Chloe Robinson, Lia Panman, Ioannis Kasioulis, 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.
bioRxiv · Jan 21, 2025 · PreprintCell Biology