Design, Synthesis, In Vitro and In Vivo Characterization of Selective NKCC1 Inhibitors for the Treatment of Core Symptoms in Down Syndrome

Abstract excerpt

Intracellular chloride concentration [Cl–]i is defective in several neurological disorders. In neurons, [Cl–]i is mainly regulated by the action of the Na+–K+–Cl– importer NKCC1 and the K+–Cl– exporter KCC2. Recently, we have reported the discovery of ARN23746 as the lead candidate of a novel class of selective inhibitors of NKCC1. Importantly, ARN23746 is able to rescue core symptoms of Down syndrome (DS) and...

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