3025238-91-6
Chemical Structure
PTC-003
- CAS No.: 3025238-91-6
- Formula:C18H17Cl2N3OS
- Molecular Weight:394.32
InChIKey: YROLOWSZDZKJGY-RYUDHWBXSA-N
SMILES: ClC1=CC(NCC2=CC=CO2)=C3C(C(Cl)=C([C@H]4CC=CC[C@@H]4N)S3)=N1
Biological Activity: 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[1].
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PTC-003 | 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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