Fig. 7

GRN PTC readthrough rescues FTLD/NCL lysosomal pathological CSTD maturation phenotype in hiPSC-derived R493X^−/− KI cortical neurons. a DIV 80 WT and R493X^−/− KI hiPSC-derived cortical neurons were treated with vehicle solution, G418 alone, G418 in combination with CDX5–288, and rec. Human PGRN at the indicated concentrations for 72 h. Expression of mature CSTD in treated WT and R493X^−/− KI cortical neuron lysates analyzed by western blotting, using actin as the loading control. b Densitometric quantification of CSTD expression in the aforementioned cortical neuron lysates normalized to vehicle-treated WT levels. n = 3–6 independent cultures; values are shown as mean ± SEM; * p < 0.05, ** p < 0.01, was determined by one-way ANOVA with Tukey’s multiple comparison test