Targeting mitochondrial defects and oxidative stress in Pompe Disease: from pathogenesis to therapy

Project Information

MITOPOMPE

Grant agreement ID: 797144

DOI

10.3030/797144

Project closed

EC signature date 3 April 2018

Start date 1 January 2019

End date 31 December 2020

Funded under

• EXCELLENT SCIENCE - Marie Skłodowska-Curie Actions

Total cost

€ 173 076,00

EU contribution

€ 173 076,00

Coordinated by

ASSOCIATION GENETHON
France

CORDIS provides links to public deliverables and publications of HORIZON projects.

Links to deliverables and publications from FP7 projects, as well as links to some specific result types such as dataset and software, are dynamically retrieved from OpenAIRE .

Publications

Peer reviewed articles (3)

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase

Author(s): Umut Cagin, Francesco Puzzo, Manuel Jose Gomez, Maryse Moya-Nilges, Pauline Sellier, Catalina Abad, Laetitia Van Wittenberghe, Nathalie Daniele, Nicolas Guerchet, Bernard Gjata, Fanny Collaud, Severine Charles, Marcelo Simon Sola, Olivier Boyer, Jacomina Krijnse-Locker, Giuseppe Ronzitti, Pasqualina Colella, Federico Mingozzi
Published in: Molecular Therapy, Issue 28/9, 2020, Page(s) 2056-2072, ISSN 1525-0016
Publisher: Nature Publishing Group
DOI: 10.1016/j.ymthe.2020.05.025

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects

Author(s): Pasqualina Colella, Pauline Sellier, Manuel J. Gomez, Maria G. Biferi, Guillaume Tanniou, Nicolas Guerchet, Mathilde Cohen-Tannoudji, Maryse Moya-Nilges, Laetitia van Wittenberghe, Natalie Daniele, Bernard Gjata, Jacomina Krijnse-Locker, Fanny Collaud, Marcelo Simon-Sola, Severine Charles, Umut Cagin, Federico Mingozzi
Published in: EBioMedicine, Issue 61, 2020, Page(s) 103052, ISSN 2352-3964
Publisher: Elsevier BV
DOI: 10.1016/j.ebiom.2020.103052

Deep morphological analysis of muscle biopsies from type III glycogenesis (GSDIII), debranching enzyme deficiency, revealed stereotyped vacuolar myopathy and autophagy impairment

Author(s): Pascal Laforêt, Michio Inoue, Evelyne Goillot, Claire Lefeuvre, Umut Cagin, Nathalie Streichenberger, Sarah Leonard-Louis, Guy Brochier, Angeline Madelaine, Clemence Labasse, Carola Hedberg-Oldfors, Thomas Krag, Louisa Jauze, Julien Fabregue, Philippe Labrune, Jose Milisenda, Aleksandra Nadaj-Pakleza, Sabrina Sacconi, Federico Mingozzi, Giuseppe Ronzitti, François Petit, Benedikt Schoser, Anders
Published in: Acta Neuropathologica Communications, Issue 7/1, 2019, ISSN 2051-5960
Publisher: BioMed Central
DOI: 10.1186/s40478-019-0815-2