Bioactivity | 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]. | ||||||||||||
Name | BPN-15477 | ||||||||||||
CAS | 1971086-99-3 | ||||||||||||
Formula | C12H10ClN5 | ||||||||||||
Molar Mass | 259.69 | ||||||||||||
Appearance | Solid | ||||||||||||
Transport | Room temperature in continental US; may vary elsewhere. | ||||||||||||
Storage |
|
||||||||||||
Reference | [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. |