repurposing ciclopirox as a pharmacological chaperone in a model of congenital erythropoietic porphyria

• DOI: 10.5281/zenodo.3606660
• Publication Date: 2018-09-19
• Authors: Pedro Urquiza; Ana Laín; Arantza Sanz-Parra; Jorge Moreno; Ganeko Bernardo-S...; Pierre Dubus; Esperanza González; Virginia Gutiérre...; Sandra García; Hasier Eraña; Itxaso San Juan; Iratxe Macías; Fredj Ben Bdira; Paula Pluta; Gabriel Ortega; Julen Oyarzábal; Rosario González-...; Juan Rodríguez-Cu...; Juan Anguita; Emilio Díez; Jean-Marc Blouin; Hubert de Verneuil; José M. Mato; Emmanuel Richard; Juan M. Falcón-Pérez; Joaquín Castilla; Oscar Millet
• Keywords: 0303 health sciences; Porphyria, Erythropoietic; Drug Repositioning; Ciclopirox; Uroporphyrinogen III Synthetase; Biophysical Phenomena; Cell Line; 3. Good health; Disease Models, Animal; Mice; 03 medical and health sciences; Phenotype; Animals; Homeostasis; Allosteric Site

The off-patent marketed antifungal ciclopirox improves symptoms in a mouse model of congenital erythropoietic porphyria.