Repurposing ciclopirox as a pharmacological chaperone in a model of congenital erythropoietic porphyria-Reference-Cited by-同舟云学术

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

Published:2018-09-19 Issue:459 Volume:10 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Urquiza Pedro¹^ORCID,Laín Ana¹,Sanz-Parra Arantza¹,Moreno Jorge²,Bernardo-Seisdedos Ganeko³^ORCID,Dubus Pierre⁴⁵,González Esperanza⁶^ORCID,Gutiérrez-de-Juan Virginia⁷,García Sandra³,Eraña Hasier³,San Juan Itxaso¹,Macías Iratxe¹,Ben Bdira Fredj¹⁸^ORCID,Pluta Paula¹,Ortega Gabriel¹⁹^ORCID,Oyarzábal Julen¹⁰^ORCID,González-Muñiz Rosario¹¹,Rodríguez-Cuesta Juan¹²,Anguita Juan¹²¹³¹⁴^ORCID,Díez Emilio³,Blouin Jean-Marc¹⁵,de Verneuil Hubert¹⁵,Mato José M.⁷¹⁶,Richard Emmanuel¹⁵^ORCID,Falcón-Pérez Juan M.⁶¹³¹⁶,Castilla Joaquín²¹³,Millet Oscar¹^ORCID

Affiliation:

1. Protein Stability and Inherited Disease Laboratory, CIC bioGUNE, 48160 Derio, Spain.

2. Prion Research Laboratory, CIC bioGUNE, 48160 Derio, Spain.

3. Atlas Molecular Pharma S. L., 48160 Derio, Spain.

4. Univerité de Bordeaux, Bordeaux Research in Translational Oncology, INSERM U1053, F-33000 Bordeaux, France.

5. INSERM, Biothérapie des Maladies Génétiques, Inflammatoires et Cancers, U1035, Bordeaux, France.

6. Exosomes Laboratory, CIC bioGUNE, 48160 Derio, Spain.

7. Liver Metabolism Laboratory, CIC bioGUNE, 48160 Derio, Spain.

8. Department of Macromolecular Biochemistry, Leiden Institute of Chemistry, 2300 RA Leiden, Netherlands.

9. Department of Chemistry and Biochemistry, University of California, Santa Barbara, Santa Barbara, CA 93106–9510, USA.

10. Small Molecule Discovery Platform, Center for Applied Medical Research, University of Navarra, 31008 Pamplona, Spain.

11. Instituto de Química Médica, IQM-CSIC, 28006 Madrid, Spain.

12. Animal Facility, CIC bioGUNE, 48160 Derio, Spain.

13. Ikerbasque, Basque Foundation for Science, 48013 Bilbao, Spain.

14. Macrophage and Tick Vaccine Laboratory, CIC bioGUNE, 48160 Derio, Spain.

15. Université de Bordeaux, Biothérapie des Maladies Génétiques, Inflammatoires et Cancers, U1035, F-33000 Bordeaux, France.

16. CIBERehd-ISCiii, 28029 Madrid, Spain.

Abstract

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

Funder

MINECO

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference47 articles.

1. The porphyrias: advances in diagnosis and treatment

2. Congenital erythropoietic porphyria: advances in pathogenesis and treatment

3. Structural, thermodynamic, and mechanistical studies in uroporphyrinogen III synthase:Molecular basis of congenital erythropoietic porphyria

4. Congenital erythropoietic porphyria

5. Cutaneous Porphyrias: Causes, Symptoms, Treatments and the Danish Incidence 1989–2013

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