Article
Rescue of functional CFTR channels in cystic fibrosis: a dramatic multivalent effect using iminosugar cluster-based correctors.
Chembiochem : a European journal of chemical biology - 11 Oct 2013
Compain Philippe, Decroocq Camille, Joosten Antoine, de Sousa Julien, Rodríguez-Lucena David, Butters Terry D, Bertrand Johanna, Clément Romain, Boinot Clément, Becq Frédéric, Norez Caroline
Abstract excerpt
Cystic fibrosis is caused by a mutation in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. N-butyl 1-deoxynojirimycin (N-Bu DNJ), a clinical candidate for the treatment of cystic fibrosis, is able to act as a CFTR corrector by overcoming the processing defect of the mutant protein. To explore the potential of multivalency on CFTR correction activity, a library of twelve DNJ...
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