NVS-SM2,99.00%

产品编号:Bellancom-111520| CAS NO:1562333-92-9| 分子式:C23H30N6O| 分子量:406.52

本网站销售的所有产品仅用于工业应用或者科学研究等非医疗目的,不可用于人类或动物的临床诊断或者治疗,非药用,非食用,

货号 包装 价格 库存与货期 购买量 操作
Bellancom-111520
5800.00 杭州 北京(现货)
Bellancom-111520
9400.00 杭州 北京(现货)

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NVS-SM2

产品介绍 NVS-SM2 是一种有效的、具有口服活性和可透过血脑屏障的 SMN2 剪接增强剂,对 SMN 的 EC50 为 2 nM。NVS-SM2 增强 U1-pre-mRNA 关联。NVS-SM2 促进外显子 7 包含并恢复正常存活运动神经元 (SMN) 蛋白的表达。NVS-SM2 可用于脊椎肌肉萎缩 (SMA) 的研究。
生物活性

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.

体外研究

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.

西域 has not independently confirmed the accuracy of these methods. They are for reference only.

体内研究

NVS-SM2 (0.1-1 mg/kg; s.c.; for 30 days) treatment extends survival in a severe SMA mouse model.
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.

西域 has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: Severe SMA mice
Dosage: 0.1 mg/kg and 1 mg/kg
Administration: Subcutaneous injection; daily from day 2 to day 15, followed by every other day until day 30
Result: Extended survival in a severe SMA mouse model.
体内研究

NVS-SM2 (0.1-1 mg/kg; s.c.; for 30 days) treatment extends survival in a severe SMA mouse model.
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.

西域 has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: Severe SMA mice
Dosage: 0.1 mg/kg and 1 mg/kg
Administration: Subcutaneous injection; daily from day 2 to day 15, followed by every other day until day 30
Result: Extended survival in a severe SMA mouse model.
性状Solid
溶解性数据
In Vitro: 

DMSO : 75 mg/mL (184.49 mM; Need ultrasonic)

配制储备液
浓度 溶剂体积 质量 1 mg 5 mg 10 mg
1 mM 2.4599 mL 12.2995 mL 24.5990 mL
5 mM 0.4920 mL 2.4599 mL 4.9198 mL
10 mM 0.2460 mL 1.2300 mL 2.4599 mL
*

请根据产品在不同溶剂中的溶解度选择合适的溶剂配制储备液;一旦配成溶液,请分装保存,避免反复冻融造成的产品失效
储备液的保存方式和期限:-80°C, 6 months; -20°C, 1 month。-80°C 储存时,请在 6 个月内使用,-20°C 储存时,请在 1 个月内使用。

运输条件

Room temperature in continental US; may vary elsewhere.

储存方式
Powder -20°C 3 years
In solvent -80°C 6 months
-20°C 1 month
参考文献

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