Catalog No.: PC-25277Not For Human Use, Lab Use Only.
PTC-003 is a potent splicing modulator compound (SMC) that correct microtubule-associated protein tau (MAPT) splicing with EC2x value of 5.2 nM in MAPT minigene assay in HEK293T cells (3R MAPT), promotes MAPT exon 10 exclusion, increasing 3R MAPT transcript and protein levels.
PTC-003 is a potent splicing modulator compound (SMC) that correct microtubule-associated protein tau (MAPT) splicing with EC2x value of 5.2 nM in MAPT minigene assay in HEK293T cells (3R MAPT), promotes MAPT exon 10 exclusion, increasing 3R MAPT transcript and protein levels.
PTC-003 induces a dose-dependent increase in 3R tau in MSD protein assay EC1.3X value of 7.4 nM.
PTC-003 lowers 4R MAPT expression and rescues tauopathy phenotypes in the S305N iPSC-neuronal model.
Neuronal pre- treatment with PTC-003 also significantly protected neurons against stress conditions.
PTC-003 (2.5 mg/kg via oral gavage) ameliorates tau pathology in the human tau-N279K gene-replacement mouse model, reduces pTau231 in the brain.
PTC-003 promotes MAPT exon 10 skipping, increasing 3R MAPT transcript and protein levels in the brain as a consequence of reduced 4R MAPT.
