SMN-C3 |
رقم الكتالوجGC30185 |
SMN-C3 هو معدل SMN2 النشط عن طريق الفم ولديه القدرة على علاج ضمور العضلات الشوكي (SMA)
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Cas No.: 1449597-34-5
Sample solution is provided at 25 µL, 10mM.
SMN-C3 is an orally active SMN2 splicing modulator and has the potential to treat spinal muscular atrophy (SMA).
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[1].
[1]. Naryshkin NA, et al. Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy. Science. 2014 Aug 8;345(6197):688-93.
Animal experiment: | Mice[1]The animals are treated with SMN-C3 at doses of 0.3, 1, and 3 mg/kg per day by intraperitoneal injections from P3 through P23 and thereafter at doses of 1, 3, and 10 mg/kg per day, respectively, by oral gavage[1]. |
References: [1]. Naryshkin NA, et al. Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy. Science. 2014 Aug 8;345(6197):688-93. |
Cas No. | 1449597-34-5 | SDF | |
Canonical SMILES | O=C1C=C(C2=NN3C(C(C)=NC(C)=C3)=C2)N=C4N1C=C(C5CCN(CC)CC5)C=C4C | ||
Formula | C24H28N6O | M.Wt | 416.52 |
الذوبان | DMSO : 5 mg/mL (12.00 mM);Water : < 0.1 mg/mL (insoluble) | Storage | Store at -20°C |
General tips | Please select the appropriate solvent to prepare the stock solution according to the
solubility of the product in different solvents; once the solution is prepared, please store it in
separate packages to avoid product failure caused by repeated freezing and thawing.Storage method
and period of the stock solution: When stored at -80°C, please use it within 6 months; when stored
at -20°C, please use it within 1 month. To increase solubility, heat the tube to 37°C and then oscillate in an ultrasonic bath for some time. |
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Shipping Condition | Evaluation sample solution: shipped with blue ice. All other sizes available: with RT, or with Blue Ice upon request. |
Prepare stock solution | |||
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1 mg | 5 mg | 10 mg |
1 mM | 2.4008 mL | 12.0042 mL | 24.0085 mL |
5 mM | 0.4802 mL | 2.4008 mL | 4.8017 mL |
10 mM | 0.2401 mL | 1.2004 mL | 2.4008 mL |
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Working concentration: mg/ml;
Method for preparing DMSO master liquid: mg drug pre-dissolved in μL DMSO ( Master liquid concentration mg/mL, Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug. )
Method for preparing in vivo formulation: Take μL DMSO master liquid, next addμL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O, mix and clarify.
Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.
Note: 1. Please make sure the liquid is clear before adding the next solvent.
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- Purity: >99.50%
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