产品描述
Posenacaftor (PTI-801) sodium is a cystic fibrosis transmembrane regulator (CFTR) protein modulator that corrects the folding and trafficking of CFTR protein. Posenacaftor sodium is used for the research of cystic fibrosis (CF)[1].
体外活性
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 always 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].
Cas No.
2095064-06-3
分子式
C27H27NNaO5
分子量
468.5
别名
Posenacaftor sodium
储存和溶解度
DMSO:200 mg/mL (427.82 mM),Need ultrasonic
Powder: -20°C for 3 years
In solvent: -80°C for 2 years