(R)-Posenacaftor (R)-PTI-801) sodium 是 Posenacaftor 的 R 对映异构体。Posenacaftor (PTI-801) 是一种囊性纤维化跨膜调节蛋白 (CFTR) 调节剂，可纠正 CFTR 蛋白的折叠和运输。Posenacaftor 用于囊性纤维化 (CF) 的研究。
货号:ajcx33870
CAS:2095064-09-6
分子式:C27H26NNaO5
分子量：467.49
溶解度:DMSO : 38.33 mg/mL (81.99 mM; Need ultrasonic)
纯度：98%
存储：Store at -20°C
库存：现货

Background:

(R)-Posenacaftor (R)-PTI-801) sodium is the R enantiomer of Posenacaftor. Posenacaftor is a cystic fibrosis transmembrane regulator (CFTR) protein modulator that corrects the folding and trafficking of CFTR protein. Posenacaftor is used for the research of cystic fibrosis (CF)[1].

Cystic fibrosis (CF) is an autosomal recessive disorder, caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR)[1].CFTR is a cAMP-regulated chloride channel that is primarily located at the apical membrane of epithelial cells. Mutations in the CFTR gene lead to the production of a defective and misfolded CFTR protein, and impairs the flow of ions in and out of cells[1].Posenacaftor is a CFTR corrector, correctors are designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways[2].

[1]. Benjamin Kopp, et al. Compositions et procÉdÉs pour amÉliorer la fonction cftr dans des cellules affectÉes par la fibrose kystique. Patent WO2019156946.