SRI-41315 induces a prolonged pause at stop codons and suppresses PTCs (premature termination codons) associated with cystic fibrosis in immortalized and primary human bronchial epithelial cells, restoring CFTR (cystic fibrosis transmembrane conductance regulator) expression and function. SRI-41315 suppresses PTCs by reducing the abundance of the termination factor eRF1. SRI-41315 also potentiates aminoglycoside-mediated readthrough, leading to synergistic increases in CFTR activity.
性状
Solid
体外研究(In Vitro)
SRI-41315 exhibits target cell cytotoxicity (CC50) values >50?μM in both FRT and 16BE14o- cells.SRI-41315 shows improved potency and efficacy in FRT cells that translated to 16HBE14o- cells.SRI-41315 (5 μM, 20 h) depletes eRF1 levels through a proteasome-mediated degradation pathway. has not independently confirmed the accuracy of these methods. They are for reference only.Western Blot Analysis
运输条件
Room temperature in continental US; may vary elsewhere.
储存方式
Powder -20°C 3 years;4°C 2 years
参考文献
[1]. Sharma J, et al. A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion. Nat Commun. 2021 Jul 16;12(1):4358.
溶解度数据
In Vitro: DMSO : 20.83 mg/mL (58.28 mM; Need ultrasonic)配制储备液