Publications Peer reviewed articles (4) Rescue of GSDIII phenotype with gene transfer requires liver- and muscle- targeted GDE expression Author(s): 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 Published in: Molecular Therapy, 2017, ISSN 1525-0016 Publisher: Nature Publishing Group DOI: 10.1016/j.ymthe.2017.12.019 Combination Therapy Is the New Gene Therapy? Author(s): Giuseppe Ronzitti, Federico Mingozzi Published in: Molecular Therapy, Issue 26/1, 2018, Page(s) 12-14, ISSN 1525-0016 Publisher: Nature Publishing Group DOI: 10.1016/j.ymthe.2017.12.008 Emerging Issues in AAV-Mediated In Vivo Gene Therapy Author(s): Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi Published in: Molecular Therapy - Methods & Clinical Development, Issue 8, 2018, Page(s) 87-104, ISSN 2329-0501 Publisher: Nature publishing group DOI: 10.1016/j.omtm.2017.11.007 Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase Author(s): 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 Published in: Science Translational Medicine, Issue 9/418, 2017, Page(s) eaam6375, ISSN 1946-6234 Publisher: American Association for the Advancement of Science DOI: 10.1126/scitranslmed.aam6375 Searching for OpenAIRE data... There was an error trying to search data from OpenAIRE No results available
