Fig. 2
From: A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1
Mutant NPC1 i3Neurons exhibit lysosomal dysfunction. Electron microscopy of NPC1+/+ i3Neurons (A) and NPC1−/− i3Neurons (B) was performed at 21 days post-differentiation. A Electron-dense structures (lysosomes) and characteristic lamellar bodies (lysosomal storage) are indicated by white and black arrowheads, respectively. Scale bar, 1 μm. B Mitochondria are indicated by white arrowheads in both NPC1+/+- and NPC1−/−-differentiated neurons. Images are representative of at least three independent experiments. Scale bar, 1 μm. C Protein levels in NPC1+/+ and NPC1−/− i3Neurons 10 days post-differentiation were analyzed by western blot. The presented data is representative of three independent experiments (n = 3). D Lysosomal pH was measured in NPC1+/+ and NPC1−/− i3Neurons using dual wavelength ratio imaging. Each point represents an independent measurement, and three independent experiments are displayed using different colors. E NPC1+/+ and NPC1−/− i3Neurons were differentiated for 10 days, fixed, and stained with Magic Red (red) to assess the cathepsin B function and Hoechst (blue, nuclei). Magic Red signal intensity was plotted relative to nuclear count. In average, 100 cells were counted. The results were obtained from four independent experiments (n = 4). Scale bar, 50 μm. *p < 0.05, **p < 0.01, ****p < 0.0001 using Mann-Whitney test