Figure 4.

LDLR activity restoration upon gene correction in definitive endoderm

(A) Receptor-mediated endocytosis of labeled human low-density lipoprotein (LDL) in hiPSC-derived endoderm. The y axis represents the percentage of pHrodo Red-positive cells measured by flow cytometry after an incubation of 1 or 3 h. Unlabeled (n = 1), not edited (hiPSCs from Pt 3 and Pt 4; n = 2), edited (two independent hiPSC lines per patient, Pt 3 and Pt 4; n = 4), healthy control (two independent hiPSC lines; n = 2). Data are represented as mean + standard deviation. ^∗∗p = 0.004.

(B) Live-cell imaging illustrating LDL-pHrodo Red endocytosis. The white bar represents 100 μM.