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