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BPN-15477是一种剪接调节剂化合物 (SMC, splicing modulator compound),可恢复 ELP1(Elongator complex protein 1)外显子20的正确剪接,导致ELP1蛋白和全长转录的增加,提高包括脑在内的所有组织体内功能蛋白的水平,用于研究剪接方面遗传疾病,如家族性额颞叶痴呆(MAPT)和囊性纤维化(CFTR)等。


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BPN-15477是一种剪接调节剂化合物 (SMC, splicing modulator compound),可恢复 ELP1(Elongator complex protein 1)外显子20的正确剪接,导致ELP1蛋白和全长转录的增加,提高包括脑在内的所有组织体内功能蛋白的水平,用于研究剪接方面遗传疾病,如家族性额颞叶痴呆(MAPT)和囊性纤维化(CFTR)等。
| 规格 | 价格 | 库存 | 数量 |
|---|---|---|---|
| 1 mg | ¥ 347 | In stock | |
| 5 mg | ¥ 828 | In stock | |
| 10 mg | ¥ 1,270 | In stock | |
| 25 mg | ¥ 2,150 | In stock | |
| 50 mg | ¥ 3,180 | In stock | |
| 100 mg | ¥ 4,680 | In stock | |
| 1 mL x 10 mM (in DMSO) | ¥ 892 | In stock |
BPN-15477 相关产品
| 产品描述 | BPN-15477 is a splicing modulator compound (SMC) that which restores correct splicing of exon 20 in ELP1 (Elongator complex protein 1). This leads to increased ELP1 protein and full-length transcripts, elevating functional protein levels in all tissues including the brain. It is employed in studying splicing-related genetic disorders such as familial frontotemporal dementia (MAPT) and cystic fibrosis (CFTR). |
| 体外活性 | 方法:BPN-15477(0.3–10μM,3天)处理稳定表达剪接突变c.2988G > A的CF支气管上皮(CFBE)细胞系,通过测量处理过的CFBE细胞上的短路电流(Isc)来评估氯离子通道功能。 |
| 别名 | BPN15477 |
| 分子量 | 259.69 |
| 分子式 | C12H10ClN5 |
| CAS No. | 1971086-99-3 |
| Smiles | Clc1nc(NCc2ccncc2)c2cc[nH]c2n1 |
| 存储 | store at low temperature | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature. | ||||||||||||||||||||||||||||||
| 溶解度信息 | DMSO: 20 mg/mL (77.01 mM), Sonication is recommended. | ||||||||||||||||||||||||||||||
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DMSO
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