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, A. M. Syanda, L. Chao Zheng, M. Zieger, C. Greer, S. B. Hatch, J. M. Segal, S. J. I. Blackford, V. Chowdary, T. Ismali, D. Danovi, S. Sahdeo, D. Ebner, , 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.
bioRxiv · Sep 17, 2021 · PreprintCell Biology