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Repairing mutated proteins – development of small molecules targeting defects in the cystic fibrosis transmembrane conductance regulator

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Pages 691-708 | Published online: 11 Apr 2013

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Hong Yang & Tonghui Ma. (2015) F508del-cystic fibrosis transmembrane regulator correctors for treatment of cystic fibrosis: a patent review. Expert Opinion on Therapeutic Patents 25:9, pages 991-1002.
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Benjamin Boucherle, Johanna Bertrand, Bruno Maurin, Brice-Loïc Renard, Antoine Fortuné, Brice Tremblier, Frédéric Becq, Caroline Norez & Jean-Luc Décout. (2014) A new 9-alkyladenine-cyclic methylglyoxal diadduct activates wt- and F508del-cystic fibrosis transmembrane conductance regulator (CFTR) in vitro and in vivo. European Journal of Medicinal Chemistry 83, pages 455-465.
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Ursula E. Seidler. (2013) Gastrointestinal HCO3− transport and epithelial protection in the gut: new techniques, transport pathways and regulatory pathways. Current Opinion in Pharmacology 13:6, pages 900-908.
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Barbara Dhooghe, Sabrina Noël, Caroline Bouzin, Gaëtane Behets-Wydemans & Teresinha Leal. (2013) Correction of Chloride Transport and Mislocalization of CFTR Protein by Vardenafil in the Gastrointestinal Tract of Cystic Fibrosis Mice. PLoS ONE 8:10, pages e77314.
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