BPN-15477 是一种有效的 SMC (剪接调节剂化合物)，可恢复 ELP1 (延长蛋白复合物 1) 外显子 20 的正确剪接。BPN-15477 纠正了 ELP1 转录本的剪接，显著提高了包括大脑在内的所有组织的体内功能蛋白水平。BPN-15477 可用于额颞痴呆的研究。
货号:ajcx37578
CAS:1971086-99-3
分子式:C12H10ClN5
分子量：259.69
溶解度:N/A
纯度：98%
存储：Store at -20°C
库存：现货

Background:

BPN-15477 is a potent SMC (splicing modulator compound) that restores correct splicing of ELP1 (Elongator complex protein 1) exon 20. BPN-15477 corrects splicing of the ELP1 transcript, significantly increases the level of functional protein in vivo in all tissues, including brain. BPN-15477 can be used for frontotemporal dementia research[1].

[1]. Gao D, et al. A deep learning approach to identify gene targets of a therapeutic for human splicing disorders. Nat Commun. 2021 Jun 7;12(1):3332.