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Genetic correction of glycogen debranching enzyme deficiency in Glycogen Storage disease III: a proof of concept study

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Publications

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
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
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
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
DOI: 10.1126/scitranslmed.aam6375