Fig. 8
Effect of DT, HPBCD and enzyme replacement therapy on the accumulation of subunit c in patient NSCs. Cells were treated with 20 μM DT, 1 mM HPBCD, 20 μM DT plus 125 μM HPBCD, or 200 nM rPPT1/rTPP1 for 3 days. PPT1/TPP1 expression were restored after the PPT1/TPP1 replacement therapy. After the treatment with 20 μM DT plus 125 μM HPBCD, the expression of subunit c in PPT1E8/E1NSCs decreased by 75% (a and b). Moreover, it showed that subunit c expression in TPP1E4/E6 NSCs and TPP1E4/IVS5 NSCs was decreased by 51% and 64% respectively, in TPP1 replacement treatment, and also decreased by 18%, 30% with DT treatment (c, d, e and f). Data are displayed as mean ± SD. * P < 0.05, ** P < 0.01