Branaplam (LMI070) (LMI070; NVS-SM1) 是一种高效、选择性和口服活性的存活运动神经元 2 (SMN2) 剪接调节剂，对 SMN 的 EC50 为 20 nM。 Branaplam (LMI070) 抑制人类-ether-a-go-go-相关基因 (hERG)，IC50 为 6.3 μM. Branaplam (LMI070) 提升全长 SMN 蛋白并延长严重脊髓性肌萎缩 (SMA) 小鼠模型的存活时间。
| Cas No. | 1562338-42-4 |
| 别名 | LMI070; NVS-SM1 |
| Canonical SMILES | OC1=CC(C2=CNN=C2)=CC=C1C3=CC=C(OC4CC(C)(C)NC(C)(C)C4)N=N3 |
| 分子式 | C22H27N5O2 |
| 分子量 | 393.48 |
| 溶解度 | DMSO : 6.12 mg/mL (15.55 mM) |
| 储存条件 | Store at -20°C |
| General tips | For obtaining a higher solubility , please warm the tube at 37 ℃ and shake it in the ultrasonic bath for a while. |
| Shipping Condition | Evaluation sample solution : ship with blue ice
All other available size: ship with RT , or blue ice upon request |
| 产品描述 | Branaplam (LMI070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator. [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. |
