产品说明书

Branaplam

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Chemical Structure| 1562338-42-4 同义名 : LMI070;NVS-SM1
CAS号 : 1562338-42-4
货号 : A758913
分子式 : C22H27N5O2
纯度 : 99%+
分子量 : 393.482
MDL号 : MFCD29472288
存储条件：

粉末 Keep in dark place,Sealed in dry,Store in freezer, under -20°C

液体 -20°C:3-6个月-80°C:12个月
溶解度 :

DMSO: 30 mg/mL(76.24 mM)，配合低频超声，并水浴加热至45℃助溶，注意：DMSO长时间开封后，会吸水并导致溶解能力下降，请避免使用长期开封的DMSO
动物实验配方：
生物活性
描述 Branaplam, identified as LMI070 or NVS-SM1, is distinguished by its potent, selective, and orally bioavailable qualities as an SMN2 splicing modulator, displaying an EC50 of 20 nM. It inhibits the hERG channel with an IC50 of 6.3 μM, enhances full-length SMN protein levels, and improves survival in a severe spinal muscular atrophy (SMA) mouse model[1].[2].
实验方案
1mg 5mg 10mg

1 mM

5 mM

10 mM

2.54mL

0.51mL

0.25mL

12.71mL

2.54mL

1.27mL

25.41mL

5.08mL

2.54mL
参考文献

[1]Palacino J, et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat Chem Biol. 2015 Jul;11(7):511-517.

[2]Cheung AK, et al. Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Dec 27;61(24):11021-11036.