[CAS NO. 2476724-74-8] PTC258
PRODUCTS SPECIFICATIONS [2476724-74-8]
DESCRIPTION [2476724-74-8]
Overview
| MDL | - |
|---|---|
| Molecular Weight | 351.92 |
| Molecular Formula | C16H18ClN3S2 |
| SMILES | CC(C1=NC(Cl)=CC(NCC2=CC=CS2)=C1S3)=C3C[C@H](C)N |
Summary
PTC258 is a specific and orally active splicing modulator of Elongator complex protein 1 gene (ELP1). PTC258 increases the expression of ELP1 in vitro and in vivo. PTC258 is well tolerated in mouse model [1] .
In Vitro
PTC258 (0.01 nM-0.01 μM; 48 h) efficiently increases full-length ELP1 mRNA and protein in Familial Dysautonomia (FD) patient fibroblasts [1] .
MCE has not independently confirmed the accuracy of these methods. They are for reference only.
In Vivo
PTC258 (3-24 mg/kg; p.o.; once daily for 3 months) is well tolerated. It corrects the splicing of the ELP1 transcript and significantly increases the amount of functional protein in vivo in all tissues tested, including the brain in mouse model [1] .
MCE has not independently confirmed the accuracy of these methods. They are for reference only.
| Animal Model: | Familial Dysautonomia (FD) Mouse model [1] |
| Dosage: | 3 mg/kg, 6 mg/kg, 12 mg/kg, 24 mg/kg |
| Administration: | Oral gavage; once daily for 3 month |
| Result: | Increased full-length ELP1 transcript in a dose-dependent manner. And also, it increased in functional ELP1 protein in the brain, trigeminal, liver, and quadricep. |
Shipping
Room temperature in continental US; may vary elsewhere.
Storage
Please store the product under the recommended conditions in the Certificate of Analysis.