Fig. 7: The therapeutic effect of VPA is dependent on autophagic signaling. | Cell Death & Disease

Fig. 7: The therapeutic effect of VPA is dependent on autophagic signaling.

From: Human iNSC-derived brain organoid model of lysosomal storage disorder in Niemann–Pick disease type C

Fig. 7: The therapeutic effect of VPA is dependent on autophagic signaling.

a Western blot analysis and b quantification of the LC3-II and p62 levels in the WT, NPC, and VPA-treated NPC organoids. n = 3 per group. c Cholesterol levels in each organoid were quantified and normalized to those in the WT organoids. n = 10 per group. d Whole-mount filipin staining of the WT, NPC, and VPA-treated NPC organoids on day 28. Scale bars, 500 μm. e Immunostaining for NPC1 in the WT, NPC, and VPA-treated organoids. f Quantification of NPC1 staining in each group. Scale bars, 50 μm. Zoom scale bars, 10 μm. g Western blot analysis of NPC1 marker and h quantification of each group. n = 3 per group. i, j Heat map of the differentially expressed genes related to cholesterol transport and autophagy in the NPC and VPA-treated NPC organoids. n = 3 per group. *p < 0.05 and ***p < 0.001. The results are the mean ± SD.

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