NVS-SM2

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NVS-SM2

本产品不向个人销售，仅用作科学研究，不用于任何人体实验及非科研性质的动物实验。

CAS NO:

1562333-92-9

包装与价格：

包装	价格(元)
10 mM * 1 mL in DMSO	电议
5mg	电议
10mg	电议
50mg	电议
100mg	电议

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-penetrantSMN2 splicingenhancer with anEC₅₀of 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^[1]^[2].

IC₅₀& Target

EC50: 5 nM (SMN)^[1]

体外研究
(In Vitro)

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].

体内研究
(In Vivo)

NVS-SM2 (0.1-1 mg/kg; s.c.; for 30 days) treatment extends survival in a severe SMA mouse model^[2].
Pharmacokinetic analysis demonstrate that NVS-SM2 is readily available in the brain after IV and oral (PO) administration in mouse and rat with T_maxof 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].

Animal Model:	Severe SMA mice^[2]
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.

分子量

406.52

性状

Solid

Formula

C₂₃H₃₀N₆O

CAS 号

1562333-92-9

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

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℃, 6 months; -20℃, 1 month。-80℃ 储存时，请在 6 个月内使用，-20℃ 储存时，请在 1 个月内使用。