Targeting a genetic defect: cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis

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Targeting a genetic defect: cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis.

Cystic fibrosis (CF) is caused by genetic mutations that affect the cystic fibrosis transmembrane conductance regulator (CFTR) protein. These mutations can impact the synthesis and transfer of the CFTR protein to the apical membrane of epithelial cells, as well as influencing the gating or conductance of chloride and bicarbonate ions through the channel. CFTR dysfunction results in ionic imbala...

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Cystic Fibrosis Transmembrane Conductance Regulator

Description The cystic fibrosis transmembrane regulator (CFTR) gene codes for the CFTR protein; a chloride channel protein that helps in the transportation of chloride ions and water molecules across the cell membranes of lungs, liver, pancreas, and skin. CFTR is a member of the ATP-binding cassette family of membrane transport proteins, but appears to be unique within this family by functionin...

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Cystic Fibrosis Transmembrane Conductance Regulator

The cystic fibrosis transmembrane conductance regulator (CFTR) Cl channel exhibits lyotropic anion selectivity. Anions that are more readily dehydrated than Cl exhibit permeability ratios (P(S)/P(Cl)) greater than unity and also bind more tightly in the channel. We compared the selectivity of CFTR to that of a synthetic anion-selective membrane [poly(vinyl chloride)-tridodecylmethylammonium chl...

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Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives

Mutations of the CFTR gene cause cystic fibrosis (CF), the most common recessive monogenic disease worldwide. These mutations alter the synthesis, processing, function, or half-life of CFTR, the main chloride channel expressed in the apical membrane of epithelial cells in the airway, intestine, pancreas, and reproductive tract. Lung disease is the most critical manifestation of CF. It is charac...

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Prevalence of Cystic Fibrosis Trans-membrane Conductance Regulator Gene common mutations in children with cystic fibrosis in Isfahan, Iran

Background: Cystic fibrosis (CF) is the most common lethal genetic disorder of Cystic Fibrosis Trans-membrane Conductance (CFTR) Regulator gene mutations. We aimed to investigate common mutations in CF patients and to assess its possible relationship with clinical presentations. Materials and Methods: This cross sectional study was conducted on 36 CF patients who were referred to a tertiary ped...

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ژورنال

عنوان ژورنال: European Respiratory Review

سال: 2013

ISSN: 0905-9180,1600-0617

DOI: 10.1183/09059180.00008412