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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].

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].

[1]. Benjamin Kopp, et al. Compositions et procédés pour améliorer la fonction cftr dans des cellules affectées par la fibrose kystique. Patent WO2019156946.

[2]. Posenacaftor (PTI-801)

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