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PTC258 is a specific, orally active modulator of the splicing of the Elongator complex protein 1 gene (ELP1), enhancing its expression both in vitro and in vivo. Additionally, PTC258 has demonstrated good tolerability in a mouse model [1].

| Pack Size | Price | USA Warehouse | Global Warehouse | Quantity |
|---|---|---|---|---|
| 5 mg | Inquiry | 8-10 weeks | 8-10 weeks | |
| 50 mg | Inquiry | 8-10 weeks | 8-10 weeks |
| Description | PTC258 is a specific, orally active modulator of the splicing of the Elongator complex protein 1 gene (ELP1), enhancing its expression both in vitro and in vivo. Additionally, PTC258 has demonstrated good tolerability in a mouse model [1]. |
| In vivo | PTC258 (administered orally at doses of 3-24 mg/kg once daily for three months) demonstrates good tolerability. It effectively amends the splicing of the ELP1 transcript and substantially enhances the levels of the resultant functional protein across various tissues, brain included, in a mouse model [1]. |
| Molecular Weight | 351.92 |
| Formula | C16H18ClN3S2 |
| Cas No. | 2476724-74-8 |
| Smiles | N(CC1=CC=CS1)C2=C3C(C(C)=C(C[C@H](C)N)S3)=NC(Cl)=C2 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature. |
Dissolve 2 mg of the compound in 100 μL DMSO
to obtain a stock solution at a concentration of 20 mg/mL . If the required concentration exceeds the compound's known solubility, please contact us for technical support before proceeding.
1) Add 100 μL of the DMSO
stock solution to 400 μL PEG300
and mix thoroughly until the solution becomes clear.
2) Add 50 μL Tween 80 and mix well until fully clarified.
3) Add 450 μL Saline,PBS or ddH2O
and mix thoroughly until a homogeneous solution is obtained.
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