Rescue of GSDIII phenotype with gene transfer requires liver- and muscle- targeted GDE expression

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Authors: Patrice Vidal, Serena Pagliarani, Pasqualina Colella, Helena Costa Verdera, Louisa Jauze, Monika Gjorgjieva, Francesco Puzzo, Solenne Marmier, Fanny Collaud, Marcelo Simon Sola, Severine Charles, Sabrina Lucchiari, Laetitia van Wittenberghe, Alban Vignaud, Bernard Gjata, Isabelle Richard, Pascal Laforet, Edoardo Malfatti, Gilles Mithieux, Fabienne Rajas, Giacomo P. Comi, Giuseppe Ronzitti, Federic

Journal title: Molecular Therapy

Journal publisher: Nature Publishing Group

Published year: 2017

DOI identifier: 10.1016/j.ymthe.2017.12.019

ISSN: 1525-0016