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编 号:F748857
分子式:C27H27NO5
分子量:445.51
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生物活性:
Posenacaftor (PTI-801) 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).

体外研究:
Cystic fibrosis (CF) is an autosomal recessive disorder, caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR).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.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.
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