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Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.
Vidal, Patrice; Pagliarani, Serena; Colella, Pasqualina; Costa Verdera, Helena; Jauze, Louisa; Gjorgjieva, Monika; Puzzo, Francesco; Marmier, Solenne; Collaud, Fanny; Simon Sola, Marcelo; Charles, Severine; Lucchiari, Sabrina; van Wittenberghe, Laetitia; Vignaud, Alban; Gjata, Bernard; Richard, Isabelle; Laforet, Pascal; Malfatti, Edoardo; Mithieux, Gilles; Rajas, Fabienne; Comi, Giacomo Pietro; Ronzitti, Giuseppe; Mingozzi, Federico.
Afiliación
  • Vidal P; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.
  • Pagliarani S; Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Neurology Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy.
  • Colella P; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France; Genethon, 91002 Evry, France.
  • Costa Verdera H; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.
  • Jauze L; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France; Genethon, 91002 Evry, France.
  • Gjorgjieva M; Université Lyon 1, Villeurbanne 69622, France.
  • Puzzo F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France; Genethon, 91002 Evry, France.
  • Marmier S; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.
  • Collaud F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France; Genethon, 91002 Evry, France.
  • Simon Sola M; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.
  • Charles S; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France; Genethon, 91002 Evry, France.
  • Lucchiari S; Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Neurology Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy.
  • van Wittenberghe L; Genethon, 91002 Evry, France.
  • Vignaud A; Genethon, 91002 Evry, France.
  • Gjata B; Genethon, 91002 Evry, France.
  • Richard I; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France; Genethon, 91002 Evry, France.
  • Laforet P; Myology Institute, Neuromuscular Morphology Unit, Groupe Hospitalier Universitaire La Pitié-Salpêtrière, Sorbonne Universités UPMC Univ Paris 06, 75005 Paris, France; Paris-Est neuromuscular center, Pitié-Salpêtrière Hospital, APHP, 75005 Paris, France; Raymond Poincaré Teaching Hospital, APHP, 9238
  • Malfatti E; Myology Institute, Neuromuscular Morphology Unit, Groupe Hospitalier Universitaire La Pitié-Salpêtrière, Sorbonne Universités UPMC Univ Paris 06, 75005 Paris, France.
  • Mithieux G; Institut National de la Santé et de la Recherche Médicale, U1213, Lyon 69008, France; Université Lyon 1, Villeurbanne 69622, France.
  • Rajas F; Institut National de la Santé et de la Recherche Médicale, U1213, Lyon 69008, France; Université Lyon 1, Villeurbanne 69622, France.
  • Comi GP; Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Neurology Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy.
  • Ronzitti G; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France; Genethon, 91002 Evry, France. Electronic address: gronzitti@genethon.fr.
  • Mingozzi F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France; Genethon, 91002 Evry, France. Electronic address: fmingozzi@genethon.fr.
Mol Ther ; 26(3): 890-901, 2018 03 07.
Article en En | MEDLINE | ID: mdl-29396266
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Fenotipo / Terapia Genética / Sistema de la Enzima Desramificadora del Glucógeno / Enfermedad del Almacenamiento de Glucógeno Tipo III / Músculo Esquelético / Hígado Tipo de estudio: Diagnostic_studies / Prognostic_studies Límite: Animals Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2018 Tipo del documento: Article País de afiliación: Francia Pais de publicación: Estados Unidos
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Fenotipo / Terapia Genética / Sistema de la Enzima Desramificadora del Glucógeno / Enfermedad del Almacenamiento de Glucógeno Tipo III / Músculo Esquelético / Hígado Tipo de estudio: Diagnostic_studies / Prognostic_studies Límite: Animals Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2018 Tipo del documento: Article País de afiliación: Francia Pais de publicación: Estados Unidos