Posenacaftor (PTI-801) is acystic fibrosis transmembrane regulator (CFTR) proteinmodulator 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 theCFTRgene 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].

445.51

C₂₇H₂₇NO₅

2095064-05-2

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