PTC-003 | MAPT splicing modulator | Probechem Biochemicals

Chemical Structure : PTC-003

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PTC-003 (PTC003)

货号: PC-25277Not For Human Use, Lab Use Only.

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生物&药学活性

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.

物理化学性质&存储条件

分子量
分子式
外观性状	Solid
CAS No.
储存条件	固体粉末 -20°C 12 个月; 4°C 6 个月	配置液 -80°C 6 个月; -20°C 6 个月
Shipping
Solubility	10 mM in DMSO
Chemical Name/SMILES

参考文献

1. Silva MC, et al. bioRxiv [Preprint]. 2025 May 10:2025.05.06.652428.

PTC-003 (PTC003)

生物&药学活性

物理化学性质&存储条件

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PTC-003 (PTC003)

生物&药学活性

物理化学性质&存储条件

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