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Posenacaftor sodium

產(chǎn)品活性：Posenacaftor (PTI-801) sodium 是一種囊性纖維化跨膜調(diào)節(jié)蛋白 (CFTR) 調(diào)節(jié)劑，可糾正 CFTR 蛋白的折疊和運輸。Posenacaftor sodium 用于囊性纖維化 (CF) 的研究。

研究領域：Membrane Transporter/Ion Channel

作用靶點：CFTR

In Vitro: 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 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.

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