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Fig. 3 | BMC Biology

Fig. 3

From: A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1

Fig. 3

HPβCD reduces unesterified cholesterol accumulation and improves mitochondrial function in NPC1−/− i3Neurons. A Unesterified cholesterol accumulation was decreased by treatment with HPβCD. NPC1−/− i3Neurons were differentiated for 10 days and treated with 0–1000 μM hydroxypropyl-β-cyclodextrin (HPβCD) for 24 h. Cells were fixed and stained with PFO-488 (green). The nuclei were counter-stained with Hoechst (blue). At least 2000 cells were counted, and the results were obtained from five independent experiments (n = 5). Scale bar, 10 μm. BE NPC1−/− i3Neurons have impaired mitochondrial function that is partially corrected by treatment with HPβCD. NPC1+/+ and NPC1−/− i3Neurons were differentiated for 3 days and seeded in a Seahorse XF96 plate and differentiated for another 7 days. NPC1 i3Neurons were treated with and without 100 μM HPBCD (CD) for 24 h prior to analysis with the Seahorse XF Analyzer to measure the oxygen consumption rate (B); extracellular acidification rate (C); basal respiration, maximal respiration, ATP production, and spare respiration capacity (D); and energy phenotype profile (E). Individual inhibitors oligomycin A (O), FCCP (carbonyl cyanide-4-trifluoromethoxyphenylhydrazone) (F), Ant A-Rot (A) are added in each time point (n = 8 for each group). *p < 0.05, **p < 0.01, ****p < 0.0001 by non-parametric one-way ANOVA

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