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

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Authors: 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

Journal title: EBioMedicine

Journal number: 61

Journal publisher: Elsevier BV

Published year: 2020

Published pages: 103052

DOI identifier: 10.1016/j.ebiom.2020.103052

ISSN: 2352-3964