Design, Synthesis, <i>In Vitro</i> and <i>In Vivo</i> Characterization of Selective NKCC1 Inhibitors for the Treatment of Core Symptoms in Down Syndrome
Marco Borgogno, Annalisa Savardi, Jacopo Manigrasso, Alessandra Turci, Corinne Portioli, Giuliana Ottonello, Sine Mandrup Bertozzi, Andrea Armirotti, Andrea Contestabile, Laura Cancedda, Marco De Vivo
Abstract
efficacy in a mouse model of DS. These results further strengthen the potential of this new class of compounds for the treatment of core symptoms of brain disorders characterized by the defective NKCC1/KCC2 expression ratio.
Topics & Concepts
In vivoChemistryIn vitroIntracellularAutismStructure–activity relationshipCore (optical fiber)PharmacologyBiochemistryPsychologyMedicineBiologyGeneticsPsychiatryMaterials scienceComposite materialGenetics and Neurodevelopmental DisordersDown syndrome and intellectual disability researchSignaling Pathways in Disease