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USE OF N-ALKANOLS FOR THE ACTIVATION OF CFTR |
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Principal Investigator: Bernard VERRIER
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TECHNICAL DESCRIPTION
Cystic Fibrosis (CF) is a fatal autosomal recessive disease characterized by altered composition of epithelial secretions caused by mutations in the CF gene encoding the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein, a cAMP-dependant chlorure channel located in the apical membrane of epithelial cells.
In CF cells the mutant protein is mislocalized and presents a reduced activity. The present invention relates to a novel utilization of n-alkanols for the activation of CFTR. Alkanols, already known as anesthetic coumpounds, are able to activate CFTR in a specific manner by a cAMP-independant pathway and present no side-effect.
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