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

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Authors: 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

Journal title: Molecular Therapy

Journal number: 28/9

Journal publisher: Nature Publishing Group

Published year: 2020

Published pages: 2056-2072

DOI identifier: 10.1016/j.ymthe.2020.05.025

ISSN: 1525-0016