Fig. 6.

Effect of drugs on expression level of SOD2 and DHCR24. A, Human NPC1^WT and NPC1^I1061T fibroblasts were treated with DMSO or 10 μm of SAHA for 72 h. Cell lysates were subjected to SDS-PAGE, followed by Western blotting with primary antibodies against NPC1 and tubulin proteins. B, Human NPC1^WT fibroblast was treated with DMSO and NPC1^I1061T fibroblast with DMSO or seven drugs of four different categories—cyclodextrins: MβCD (0.5 mm) and HPCD (0.5 mm); HDACIs: CI-994 (10 μm), SAHA (10 μm), and VPA (4 mm); antioxidant: NAC (5 mm); and an oxysterol derivative pharmacological chaperone: mo56HC (10 μm) for 72 h. Cell lysates were immunoblotted against rabbit anti-SOD2 antibody. C, Human NPC1^WT and NPC1^I1061T fibroblasts were treated with the drugs and for the duration as mentioned above and immunoblotted against mouse anti-DHCR24 antibody. Tubulin was used as a loading control. The blots were quantified with the Image J software. In the bar graph panels, * indicates statistically significant (p < 0.05) difference in drug-treated NPC1^I1061T cells compared with DMSO-treated NPC1^I1061T cells.