Discovery of a picomolar potency pharmacological corrector of the mutant CFTR chloride channel
Nicoletta Pedemonte, Fabio Bertozzi, Emanuela Caci, Federico Sorana, Paolo Di Fruscia, Valeria Tomati, Loretta Ferrera, A. Rodríguez-Gimeno, Francesco Berti, Emanuela Pesce, Elvira Sondo, Ambra Gianotti, Paolo Scudieri, Tiziano Bandiera, Luis J. V. Galietta
Abstract
of 38 picomolar, which is more than 5000-fold lower compared to presently available corrector drugs. ARN23765 also showed high efficacy, synergy with other types of correctors, and compatibility with chronic VX-770 potentiator. Besides being a promising drug, particularly suited for drug combinations, ARN23765 represents a high-affinity probe for CFTR structure-function studies.
Topics & Concepts
MutantChloride channelPotencyChlorideChemistryProtein engineeringMutationCell biologyBiochemistryBiophysicsBiologyIn vitroEnzymeGeneOrganic chemistryCystic Fibrosis Research AdvancesInhalation and Respiratory Drug DeliveryNeonatal Respiratory Health Research