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1449597-34-5

中文名稱 1449597-34-5
英文名稱 SMN-C3
CAS 1449597-34-5
分子式 C24H28N6O
分子量 416.52
MOL 文件 1449597-34-5.mol
1449597-34-5 結(jié)構(gòu)式 1449597-34-5 結(jié)構(gòu)式

基本信息

中文別名
化合物SMN-C3
化合物 T12935
英文別名
SMN-C3
4H-Pyrido[1,2-a]pyrimidin-4-one, 2-(4,6-dimethylpyrazolo[1,5-a]pyrazin-2-yl)-7-(1-ethyl-4-piperidinyl)-9-methyl-

物理化學(xué)性質(zhì)

密度1.31±0.1 g/cm3(Predicted)
儲存條件-20°C儲存
溶解度DMSO : 5 mg/mL (12.00 mM);Water : < 0.1 mg/mL (insoluble)
酸度系數(shù)(pKa)9.27±0.10(Predicted)
形態(tài)Solid
顏色Light yellow to yellow

常見問題列表

生物活性
SMN-C3 是一種可口服的 SMN2 剪接調(diào)節(jié)劑,可用于脊椎肌肉萎縮 (SMA) 的研究。
靶點

SMN.

體內(nèi)研究

At P16, vehicle treated D7 mice are much smaller than heterozygous littermate controls and appear moribund. In contrast, D7 mice treated with the high dose of SMN-C3 show a phenotype similar to that of heterozygous controls. SMN-C3 treatment induces a dose-dependent bodyweight gain in the D7 mice, with some animals showing a body weight that is ~80% that of heterozygous controls. SMN-C3 normalizes the motor behavior of D7 mice, illustrated by the ability of the mice to right themselves as quickly as heterozygous controls and by their level of locomotor activity. Most importantly, whereas vehicle-treated mice die within 3 weeks after birth with a median survival of 18 days, SMN-C3 treatment increases survival in a dose-dependent manner to a median survival time of 28 days in the low-dose (0.3 mg/kg per day) group. In the two higher-dose groups (1 and 3 mg/kg per day), ~90% of animals survive beyond P65 when the study is completed.

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