Cell BiologybioRxivPreprint
Small molecule screen employing patient-derived iPS hepatocytes identifies LRRK2 as a novel therapeutic target for Alpha1 Antitrypsin Deficiency
D. Kent, S. S. Ng, P. Khoshkenar…·S. T. Rashid
A small molecule screen in patient iPSCs with in vivo validation in mice identifies LRRK2 as a new therapeutic target for Alpha-1 Antitrypsin Deficiency.
DOI 10.1101/2021.09.17.460732