Patient-Derived PiZZ iPSC-Hepatic Cells Exhibit Intracellular AAT Retention
(A) Schematic overview of iPSC-hepatic directed differentiation protocol. ∗p < 0.05, ∗∗p < 0.01 by one-way ANOVA.
(B) Flow cytometry of fixed, permeabilized iPSC-hepatic cells using anti-AAT and anti-FOXA1 antibodies in nine genetically distinct ESC or iPSC lines at T24.
(C) MFI of AAT and AFP in PiZZ iPSC-hepatic cells compared to WT iPSC- or ESC-hepatic cells.
(D) Quantification of AAT (SERPINA1) and AFP mRNA levels in iPSC-hepatic cells.
(E) ELISA of secreted AAT and albumin in cell supernatants at T24. ∗p < 0.05 by two-tailed t test. PS, primitive streak; DE, definitive endoderm; ACT, Activin A; Chir, Chir99021; OSM, oncostatin M. n = 3 independent experiments. Data are represented as mean ± SEM.