Synthesis and properties of molecular probes for the rescue site on mutant cystic fibrosis transmembrane conductance regulator

B Alkhouri, RA Denning, P Kim Chiaw…

Index: Alkhouri, Bashar; Denning, Robert A.; Chiaw, Patrick Kim; Eckford, Paul D.W.; Yu, Wilson; Li, Canhui; Bogojeski, Jovanka J.; Bear, Christine E.; Viirre, Russell D. Journal of Medicinal Chemistry, 2011 , vol. 54, # 24 p. 8693 - 8701

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Citation Number: 7

Abstract

Cystic fibrosis is a genetic disease caused by mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In vitro experiments have demonstrated that 4-methyl-2-(5-phenyl-1 H-pyrazol-3-yl) phenol (VRT-532, 1) is able to partially restore the function of mutant CFTR proteins. To help elucidate the nature of the interactions between 1 and mutant CFTR, molecular probes based on the structure of 1 ...