产品
编 号：F753108
分子式：C16H18ClN3S2
分子量：351.92
分子式：C16H18ClN3S2
分子量：351.92
产品类型
规格
价格
是否有货
1mg
询价
询价
5mg
询价
询价
10mg
询价
询价
结构图
CAS No: 2476724-74-8
产品详情
生物活性：
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.
体内研究：
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.Animal Model:Familial Dysautonomia (FD) Mouse model
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.
体外研究：
PTC258 (0.01 nM-0.01 μM; 48 h) efficiently increases full-length ELP1 mRNA and protein in Familial Dysautonomia (FD) patient fibroblasts.
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.
体内研究：
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.Animal Model:Familial Dysautonomia (FD) Mouse model
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.
体外研究：
PTC258 (0.01 nM-0.01 μM; 48 h) efficiently increases full-length ELP1 mRNA and protein in Familial Dysautonomia (FD) patient fibroblasts.
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