Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

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Authors: Francesco Puzzo, Pasqualina Colella, Maria G. Biferi, Deeksha Bali, Nicole K. Paulk, Patrice Vidal, Fanny Collaud, Marcelo Simon-Sola, Severine Charles, Romain Hardet, Christian Leborgne, Amine Meliani, Mathilde Cohen-Tannoudji, Stephanie Astord, Bernard Gjata, Pauline Sellier, Laetitia van Wittenberghe, Alban Vignaud, Florence Boisgerault, Martine Barkats, Pascal Laforet, Mark A. Kay, Dwight D. K

Journal title: Science Translational Medicine

Journal number: 9/418

Journal publisher: American Association for the Advancement of Science

Published year: 2017

Published pages: eaam6375

DOI identifier: 10.1126/scitranslmed.aam6375

ISSN: 1946-6234