PTC-775

PTC-775 (0-1000 nM, 24 h) promotes MAPT exon 10 skipping, reduces 4R tau, and increases 3R tau in HEK293T-MAPT minigene cells^[1].
PTC-775 increases endogenous 3R tau protein in U-87 human glioma cells^[1].
PTC-775 (0.1-1000 nM; 24 h) decreases 4R tau and increases 3R tau in P301L iPSC-derived neurons^[1].
PTC-775 (96 h and 3 weeks) reduces 4R MAPT mRNA in P301L (EC50 0.7, 1.4 nM) and S305N (EC50 1.1, 159.5 nM) iPSC-derived neurons^[1].
PTC-775 (96 h or 3 weeks) reduces 4R tau protein in P301L (EC₅₀ 5.6, 28.4 nM) and S305N (EC₅₀ 46.6, 75.8 nM) iPSC-derived neurons^[1].
PTC-775 (0.1-1000 nM (P301L); 1-10000 nM (S305N); 96 h or 3 weeks) reduces total tau and hyperphosphorylated tau species in P301L and S305N iPSC-derived neurons, by up to 80-90% at 96 h, with nearly complete reduction of oligomeric pTau S396 after 3 weeks^[1].
PTC-775 (0.1-1 μM; 96 h and 3 weeks) reduces detergent-insoluble total tau and pTau S396 in P301L and S305N iPSC-derived neurons, and nearly eliminates insoluble pTau (>80-90%) after 3 weeks^[1].

PTC-775 (0.1-1 μM; 78 h pre-incubation) rescues P301L and S305N iPSC-derived neurons from glutamate-induced death, with survival reaching 70-90% at 1 μM^[1].