PTC-775
PTC-775 is a MAPT pre-mRNA splicing modulator. PTC-775 promotes MAPT exon exclusion, reduces longer tau isoforms and increases shorter isoforms. PTC-775 reduces the accumulation of total tau and hyperphosphorylated tau species (including oligomeric and insoluble forms). PTC-775 protects neurons from glutamate-induced death. PTC-775 can be used for research on frontotemporal dementia.
For research use only. We do not sell to patients.
- CAS No.: 3025238-38-1
- Formula: C17H17Cl2N3O2S
- Molecular Weight:398.31
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Storage:
Please store the product under the recommended conditions in the Certificate of Analysis.
Biological Activity
Description
In Vitro
PTC-775 (0-1000 nM, 24 h) promotes MAPT exon 10 skipping, reduces 4R tau, and increases 3R tau in HEK293T-MAPT minigene cells[1].
PTC-775 increases endogenous 3R tau protein in U-87 human glioma cells[1].
PTC-775 (0.1-1000 nM; 24 h) decreases 4R tau and increases 3R tau in P301L iPSC-derived neurons[1].
PTC-775 (96 h and 3 weeks) reduces 4R MAPT mRNA in P301L (EC50 0.7, 1.4 nM) and S305N (EC50 1.1, 159.5 nM) iPSC-derived neurons[1].
PTC-775 (96 h or 3 weeks) reduces 4R tau protein in P301L (EC50 5.6, 28.4 nM) and S305N (EC50 46.6, 75.8 nM) iPSC-derived neurons[1].
PTC-775 (0.1-1000 nM (P301L); 1-10000 nM (S305N); 96 h or 3 weeks) reduces total tau and hyperphosphorylated tau species in P301L and S305N iPSC-derived neurons, by up to 80-90% at 96 h, with nearly complete reduction of oligomeric pTau S396 after 3 weeks[1].
PTC-775 (0.1-1 μM; 96 h and 3 weeks) reduces detergent-insoluble total tau and pTau S396 in P301L and S305N iPSC-derived neurons, and nearly eliminates insoluble pTau (>80-90%) after 3 weeks[1].
PTC-775 (0.1-1 μM; 78 h pre-incubation) rescues P301L and S305N iPSC-derived neurons from glutamate-induced death, with survival reaching 70-90% at 1 μM[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Cell Line:HEK293T-MAPT minigene cells
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Concentration:0, 0.1, 1, 4, 12, 37, 111, 333, 1000 nM
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Incubation Time:24 h
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Result:Upregulated the expression of 3R MAPT transcripts.
Chemical Information
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CAS No. 3025238-38-1
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Molecular Weight 398.31
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Formula C17H17Cl2N3O2S
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SMILES
ClC1=CC(NCC2=CC=CO2)=C3C(C(Cl)=C([C@H]4OCCC[C@@H]4N)S3)=N1
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
Please store the product under the recommended conditions in the Certificate of Analysis.
Purity & Documentation
References
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)