NVS-SM2
Based on 1 Customer Validation
NVS-SM2 is a potent, orally active and brain-penetrant SMN2 splicing enhancer with an EC50 of 2 nM for SMN. NVS-SM2 enhances U1-pre-mRNA association. NVS-SM2 promotes exon 7 inclusion and restores normal survival motor neuron (SMN) protein expression. NVS-SM2 can be used for spinal muscular atrophy (SMA) research.
商品は「研究用試薬」です。人や動物の医療用・臨床診断用・食品用の製品ではありません。
研究用途以外に使用した場合、当社は一切の責任を負いかねます。
- 純度: 99.15%
- CAS 番号: 1562333-92-9
- 分子式: C23H30N6O
- 分子量:406.52
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保管条件:Powder -20°C, 3 years ; In solvent -80°C, 6 months , -20°C, 1 month
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生物活性
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化学情報
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溶剤 & 溶解度
- 純度とドキュメンテーション
- 参考文献
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Help & FAQs
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DNA/RNA Synthesis アイソフォーム固有の製品をすべて表示
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生物活性
EC50: 5 nM (SMN)[1]
For NVS-SM2, the molecular mechanism of action is via stabilization of the transient double-strand RNA structure formed by the SMN2 pre-mRNA and U1 small nuclear ribonucleic protein (snRNP) complex. The binding affinity of U1 snRNP to the 5' splice site is increased in a sequence-selective manner, discrete from constitutive recognition[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
Pharmacokinetic analysis demonstrate that NVS-SM2 is readily available in the brain after IV and oral (PO) administration in mouse and rat with Tmax of 3 h after PO with 3 mg/kg in mice, and NVS-SM2 treatment induces a 1.5-fold increase in SMN protein levels in the mouse brain[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:Severe SMA mice[2]
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Dosage:0.1 mg/kg and 1 mg/kg
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Administration:Subcutaneous injection; daily from day 2 to day 15, followed by every other day until day 30
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Result:Extended survival in a severe SMA mouse model.
化学情報
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CAS 番号 1562333-92-9
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性状 Solid
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分子量 406.52
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分子式 C23H30N6O
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Color Off-white to light yellow
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SMILES
OC1=C(C(N=N2)=CC=C2N(C(CC3(C)C)CC(C)(N3)C)C)C=CC(C4=CNN=C4)=C1
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輸送条件
Room temperature in continental US; may vary elsewhere.
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保管条件
Powder -20°C 3 years In solvent -80°C 6 months -20°C 1 month
溶剤 & 溶解度
DMSO : 75 mg/mL (184.49 mM; Need ultrasonic; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO)
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month. When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month. When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
濃度 (開始) × 体積 (開始) = 濃度 (終了) × 体積 (終了)
純度とドキュメンテーション
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データシート (272 KB)
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SDS (251 KB)
- English - EN (251 KB)
- Français - FR (251 KB)
- Deutsch - DE (251 KB)
- Norwegian - NO (251 KB)
- Español - ES (251 KB)
- Swedish - SV (251 KB)
- Italian - IT (251 KB)
- Portuguese - PT (251 KB)
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取扱説明書 (2659 KB)
参考文献
[1]. James Palacino, et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat Chem Biol. 2015 Jul;11(7):511-7. [Content Brief]
[2]. Anne Rietz, et al. Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy. Life Sci Alliance. 2020 Nov 24;4(1):e202000889. [Content Brief]
Complete Stock Solution Preparation Table
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month. When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
| Optional Solvent | Concentration Solvent Mass | 1 mg | 5 mg | 10 mg | 25 mg |
|---|---|---|---|---|---|
| DMSO | 1 mM | 2.4599 mL | 12.2995 mL | 24.5990 mL | 61.4976 mL |
| 5 mM | 0.4920 mL | 2.4599 mL | 4.9198 mL | 12.2995 mL | |
| 10 mM | 0.2460 mL | 1.2300 mL | 2.4599 mL | 6.1498 mL | |
| 15 mM | 0.1640 mL | 0.8200 mL | 1.6399 mL | 4.0998 mL | |
| 20 mM | 0.1230 mL | 0.6150 mL | 1.2300 mL | 3.0749 mL | |
| 25 mM | 0.0984 mL | 0.4920 mL | 0.9840 mL | 2.4599 mL | |
| 30 mM | 0.0820 mL | 0.4100 mL | 0.8200 mL | 2.0499 mL | |
| 40 mM | 0.0615 mL | 0.3075 mL | 0.6150 mL | 1.5374 mL | |
| 50 mM | 0.0492 mL | 0.2460 mL | 0.4920 mL | 1.2300 mL | |
| 60 mM | 0.0410 mL | 0.2050 mL | 0.4100 mL | 1.0250 mL | |
| 80 mM | 0.0307 mL | 0.1537 mL | 0.3075 mL | 0.7687 mL | |
| 100 mM | 0.0246 mL | 0.1230 mL | 0.2460 mL | 0.6150 mL |
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