Catalog No.: PC-25280Not For Human Use, Lab Use Only.
PTC-775 is a potent splicing modulator compound (SMC) that correct microtubule-associated protein tau (MAPT) splicing with EC2x value of 4.9 nM in MAPT minigene assay in HEK293T cells (3R MAPT), promotes MAPT exon 10 exclusion, increasing 3R MAPT transcript and protein levels.
PTC-775 is a potent splicing modulator compound (SMC) that correct microtubule-associated protein tau (MAPT) splicing with EC2x value of 4.9 nM in MAPT minigene assay in HEK293T cells (3R MAPT), promotes MAPT exon 10 exclusion, increasing 3R MAPT transcript and protein levels.
PTC-775 induces a dose-dependent increase in 3R tau in MSD protein assay EC1.3X value of 8.9 nM.
PTC-775 lowers 4R MAPT expression and rescues tauopathy phenotypes in the S305N iPSC-neuronal model.
PTC-775 also significantly protected neurons against stress conditions.
