Repurposing of tamoxifen ameliorates CLN3 and CLN7 disease phenotype

Author:

Soldati Chiara1ORCID,Lopez‐Fabuel Irene234ORCID,Wanderlingh Luca G1,Garcia‐Macia Marina234ORCID,Monfregola Jlenia1,Esposito Alessandra1,Napolitano Gennaro15,Guevara‐Ferrer Marta6ORCID,Scotto Rosato Anna7,Krogsaeter Einar K7ORCID,Paquet Dominik89ORCID,Grimm Christian M7ORCID,Montefusco Sandro1,Braulke Thomas10,Storch Stephan11,Mole Sara E12,De Matteis Maria A113ORCID,Ballabio Andrea151415ORCID,Sampaio Julio L16,McKay Tristan6,Johannes Ludger16ORCID,Bolaños Juan P234ORCID,Medina Diego L15ORCID

Affiliation:

1. Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli Naples Italy

2. Institute of Functional Biology and Genomics CSIC University of Salamanca Salamanca Spain

3. Centro de Investigación Biomédica en Red sobre Fragilidad y Envejecimiento Saludable (CIBERFES) Instituto de Salud Carlos III Madrid Spain

4. Institute of Biomedical Research of Salamanca University Hospital of Salamanca CSIC University of Salamanca Salamanca Spain

5. Medical Genetics Unit Department of Medical and Translational Science Federico II University Naples Italy

6. School of Healthcare Science Manchester Metropolitan University Manchester UK

7. Faculty of Medicine Walther Straub Institute of Pharmacology and Toxicology Ludwig‐Maximilians University Munich Germany

8. Institute for Stroke and Dementia Research (ISD) University Hospital LMU Munich Munich Germany

9. Munich Cluster for Systems Neurology (SyNergy) Munich Germany

10. Department Osteology & Biomechanics University Medical Center Hamburg‐Eppendorf Hamburg Germany

11. University Children's Research@Kinder‐UKE University Medical Center Hamburg‐Eppendorf Hamburg Germany

12. Medical Research Council Laboratory for Molecular Cell Biology and UCL Great Ormond Street Institute of Child Health University College London London UK

13. Department of Molecular Medicine and Medical Biotechnology University of Napoli Federico II Naples Italy

14. Baylor College of Medicine Houston TX USA

15. Jan and Dan Duncan Neurological Research Institute Texas Children's Hospital Houston TX USA

16. Cellular and Chemical Biology Department Institut Curie, U1143 INSERM, UMR3666 CNRS PSL Research University Paris France

Funder

H2020 European Research Council

Agencia Estatal de Investigación

Plan Nacional sobre Drogas

Junta de Castilla y León

Deutsche Forschungsgemeinschaft

Medical Research Foundation

Publisher

EMBO

Subject

Molecular Medicine

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