NVS-SM2,99.00%
产品编号:Bellancom-111520| CAS NO:1562333-92-9| 分子式:C23H30N6O| 分子量:406.52
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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。 | ||||||||||||||||
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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. 西域 has not independently confirmed the accuracy of these methods. They are for reference only.
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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. 西域 has not independently confirmed the accuracy of these methods. They are for reference only.
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性状 | Solid | ||||||||||||||||
溶解性数据 |
In Vitro:
DMSO : 75 mg/mL (184.49 mM; Need ultrasonic) 配制储备液
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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。 | ||||||||||||||||
运输条件 | Room temperature in continental US; may vary elsewhere. | ||||||||||||||||
储存方式 |
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参考文献 |
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