1、Hotline: 400-820-3792Inhibitors Agonists Screening Librarieswww.MedChemESMN-C3Cat. No.: HY-112633CAS No.: 1449597-34-5分式: CHNO分量: 416.52作靶點: DNA/RNA Synthesis作通路: Cell Cycle/DNA Damage儲存式: Powder -20C 3 years4C 2 yearsIn solvent -80C 6 months-20C 1 month溶解性數據體外實驗 DMSO : 5 mg/mL (12.00 mM; Need ultra

2、sonic)H2O : 0.1 mg/mL (insoluble)Mass Solvent1 mg 5 mg 10 mg Concentration制備儲備液1 mM 2.4008 mL 12.0042 mL 24.0085 mL5 mM 0.4802 mL 2.4008 mL 4.8017 mL10 mM 0.2401 mL 1.2004 mL 2.4008 mL請根據產品在不同溶劑中的溶解度，選擇合適的溶劑配制儲備液，并請注意儲備液的保存式和期限。BIOLOGICAL ACTIVITY物活性 SMN-C3種可服的 SMN2 剪接調節劑，有治療脊椎肌萎縮 (SMA) 的潛。IC50 & Ta

3、rget SMN 1.體內研究At P16, vehicle treated D7 mice are much smaller than heterozygous littermate controls and appearmoribund. In contrast, D7 mice treated with the high dose of SMN-C3 show a phenotype similar to that of1/2 Master of Small Molecules 您邊的抑制劑師www.MedChemEheterozygous controls. SMN-C3 treatm

4、ent induces a dose-dependent bodyweight gain in the D7 mice, withsome animals showing a body weight that is 80% that of heterozygous controls. SMN-C3 normalizes themotor behavior of D7 mice, illustrated by the ability of the mice to right themselves as quickly asheterozygous controls and by their le

5、vel of locomotor activity. Most importantly, whereas vehicle-treated micedie within 3 weeks after birth with a median survival of 18 days, SMN-C3 treatment increases survival in adose-dependent manner to a median survival time of 28 days in the low-dose (0.3 mg/kg per day) group. Inthe two higher-do

6、se groups (1 and 3 mg/kg per day), 90% of animals survive beyond P65 when the study iscompleted 1.PROTOCOLAnimal Mice 1Administration 1 The animals are treated with SMN-C3 at doses of 0.3, 1, and 3 mg/kg per day by intraperitoneal injectionsfrom P3 through P23 and thereafter at doses of 1, 3, and 10

7、 mg/kg per day, respectively, by oral gavage 1.MCE has not independently confirmed the accuracy of these methods. They are for reference only.REFERENCES1. Naryshkin NA, et al. Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscularatrophy. Science. 2014 Aug 8;345(6197):688-93.McePdfHeightCaution: Product has not been fully validated for medical applications.For research