Catalog No.: PC-25278Not For Human Use, Lab Use Only.
PTC-700 is a potent splicing modulator compound (SMC) with EC2x value of 20.9 nM in MAPT minigene assay in HEK293T cells (3R MAPT), promotes MAPT exon 10 exclusion, increasing 3R MAPT transcript and protein levels.
PTC-700 is a potent splicing modulator compound (SMC) that correct microtubule-associated protein tau (MAPT) splicing with EC2x value of 20.9 nM in MAPT minigene assay in HEK293T cells (3R MAPT), promotes MAPT exon 10 exclusion, increasing 3R MAPT transcript and protein levels.
PTC-700 induces a dose-dependent increase in 3R tau in MSD protein assay EC1.3X value of 175.5 nM.
PTC-700 lowers 4R MAPT expression and rescues tauopathy phenotypes in the S305N iPSC-neuronal model.
PTC-700 also significantly protected neurons against stress conditions.
