(R)-Posenacaftor sodium (Synonyms: (R)-PTI-801 sodium) |
Catalog No.GC62513 |
Le (R)-Posénacaftor (R)-PTI-801) sodium est l'énantiomère R du Posénacaftor.
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Cas No.: 2095064-09-6
Sample solution is provided at 25 µL, 10mM.
(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.
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