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mHTT-IN-2(化合物27)作为mHTT的一种有效抑制剂,具有EC50值为0.066 μM,能够降低HTT RNA外显子[49-50]的规范剪接,从而调节HTT基因的剪接。在人类HD干细胞和BACHD小鼠模型上,mHTT-IN-2已显示出明显的体外及体内活性。此外,该化合物也被应用于研究与branaplam相关的周围神经病变。

mHTT-IN-2(化合物27)作为mHTT的一种有效抑制剂,具有EC50值为0.066 μM,能够降低HTT RNA外显子[49-50]的规范剪接,从而调节HTT基因的剪接。在人类HD干细胞和BACHD小鼠模型上,mHTT-IN-2已显示出明显的体外及体内活性。此外,该化合物也被应用于研究与branaplam相关的周围神经病变。
| 规格 | 价格 | 库存 | 数量 |
|---|---|---|---|
| 5 mg | ¥ 7,920 | 35日内发货 |
| 产品描述 | mHTT-IN-2 (compound 27) is a potent inhibitor of mutant huntingtin protein (mHTT), with an EC50 value of 0.066 μM. It specifically modulates the splicing of HTT RNA exons [49-50], acting as a splicing regulator for the HTT gene. Demonstrating inhibitory effects both in vitro and in vivo, mHTT-IN-2 has been tested in human HD stem cells and mouse BACHD models. Additionally, it is applicable in the investigation of branaplam-related peripheral neuropathy [1]. |
| 分子量 | 395.43 |
| 分子式 | C20H22FN7O |
| CAS No. | 2743336-70-9 |
| 存储 | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature. |
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