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- W2020689905 abstract "Cystic Fibrosis, one of the most common inherited lethal disease among Caucasians, is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. The CFTR protein acts as a gated Cl- channel at the apical membrane of epithelial cells, thereby facilitating proper hydration of mucosal linings. Disease causing mutations in the CFTR protein can affect a variety of steps in the biogenesis of a functional protein including the folding and trafficking of CFTR as well as the channel activity of plasma membrane-localized protein. Therefore, current research is focused on the use of small molecules to not only correct folding defects but also to enhance channel activity of mutant CFTR proteins. This review discusses the current knowledge of the folding, trafficking, and gating defects caused by CFTR mutations, the manner by which these defects are monitored by the cell, as well as the strategies which are currently being utilized to develop and screen for small molecule therapeutics. Keywords: Cystic fibrosis, chaperones, ER Quality control, correctors, potentiators" @default.
- W2020689905 created "2016-06-24" @default.
- W2020689905 creator A5015645653 @default.
- W2020689905 creator A5030361320 @default.
- W2020689905 creator A5047797660 @default.
- W2020689905 date "2009-01-01" @default.
- W2020689905 modified "2023-09-25" @default.
- W2020689905 title "The Use of Small Molecules to Correct Defects in CFTR Folding, Maturation, and Channel Activity" @default.
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