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Branaplam (LMI 070) 是一种高效、选择性和口服活性的小分子 SMN2 剪接调节剂,对 SMN 的 EC50为 20 nM。它可提高全长 SMN 蛋白并延长其生存期,也抑制 hERG,IC50为 6.3 μM。
Branaplam (LMI 070) 是一种高效、选择性和口服活性的小分子 SMN2 剪接调节剂,对 SMN 的 EC50为 20 nM。它可提高全长 SMN 蛋白并延长其生存期,也抑制 hERG,IC50为 6.3 μM。
规格 | 价格 | 库存 | 数量 |
---|---|---|---|
1 mg | ¥ 442 | 现货 | |
2 mg | ¥ 638 | 现货 | |
5 mg | ¥ 997 | 现货 | |
10 mg | ¥ 1,800 | 现货 | |
25 mg | ¥ 2,920 | 现货 | |
50 mg | ¥ 3,970 | 现货 | |
100 mg | ¥ 5,730 | 现货 | |
500 mg | ¥ 11,500 | 现货 |
产品描述 | Branaplam (LMI 070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator. |
靶点活性 | ERG (human):6.3 μM (EC50), SMN:20 nM (IC50) |
体内活性 | To evaluate the efficacy of NVS-SM1, we used the SMNΔ7 mouse model, which displays a severe phenotype. In the specific colony used for this study, death typically occurs before postnatal day 15. We were pleased to observe that, in addition to a dose-dependent elevation of SMN protein in the brain, oral administration of NVS-SM1 improved body weight and extended lifespan, with 50% of the animals in the 1 mg per kg body weight group and 62% of animals in the 3 mg per kg body weight group showing increased survival. |
别名 | LMI 070, NVS-SM1 |
分子量 | 393.48 |
分子式 | C22H27N5O2 |
CAS No. | 1562338-42-4 |
存储 | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. | |||||||||||||||
溶解度信息 | DMSO: 3.8 mg/mL (9.6 mM), Sonication and heating are recommended. | |||||||||||||||
溶液配制表 | ||||||||||||||||
DMSO
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