1 Result for "

PTC-003

" in MedChemExpress (MCE) Product Catalog:
Products (1)

1 Results for "PTC-003" in MCE Product Catalog:

Cat. No.: HY-189715
CAS No.: 3025238-91-6
Research Areas:  

Neurological Disease

PTC-003 is an orally active and blood-brain barrier-penetrant MAPT pre-mRNA splicing modulator. PTC-003 promotes skipping of exon 10 of the MAPT gene, shifting the protein isoform balance from toxic 4R-tau toward 3R-tau. PTC-003 reduces the accumulation of hyperphosphorylated tau, oligomeric tau, and insoluble tau protein. PTC-003 protects neurons from glutamate-induced neuronal stress injury, and in humanized MAPT gene-replaced N279K mice, it corrects the 3R/4R-tau isoform ratio in brain tissue and reduces the level of pathogenic pTau T231. PTC-003 can be used in research related to neurodegenerative diseases .
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