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Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

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Title: Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.
Authors: Puzzo F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.; Department of Life Sciences and Biotechnology, University of Ferrara, Ferrara, Italy., Colella P; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France., Biferi MG; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Bali D; Biochemical Genetics Laboratory, Duke University Health System, Durham, NC 27710, USA., Paulk NK; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA., 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., Collaud F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 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., Hardet R; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Leborgne C; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France., Meliani A; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Cohen-Tannoudji M; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Astord S; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Gjata B; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France., Sellier P; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., van Wittenberghe L; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France., Vignaud A; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France., Boisgerault F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France., Barkats M; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Laforet P; Paris-Est Neuromuscular Center, Pitié-Salpêtrière Hospital and Raymond Poincaré Teaching Hospital, Garches, APHP, Paris, France., Kay MA; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA., Koeberl DD; Division of Medical Genetics, Department of Pediatrics and Department of Molecular Genetics and Microbiology, Duke University School of Medicine, Durham, NC 27710, USA., Ronzitti G; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France. gronzitti@genethon.fr fmingozzi@genethon.fr., Mingozzi F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France. gronzitti@genethon.fr fmingozzi@genethon.fr.; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.
Source: Science translational medicine [Sci Transl Med] 2017 Nov 29; Vol. 9 (418).
Publication Type: Journal Article
Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE
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  Data: Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.
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  Data: <searchLink fieldCode="AU" term="%22Puzzo+F%22">Puzzo F</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.; Department of Life Sciences and Biotechnology, University of Ferrara, Ferrara, Italy.<br /><searchLink fieldCode="AU" term="%22Colella+P%22">Colella P</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.<br /><searchLink fieldCode="AU" term="%22Biferi+MG%22">Biferi MG</searchLink>; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.<br /><searchLink fieldCode="AU" term="%22Bali+D%22">Bali D</searchLink>; Biochemical Genetics Laboratory, Duke University Health System, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Paulk+NK%22">Paulk NK</searchLink>; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.<br /><searchLink fieldCode="AU" term="%22Vidal+P%22">Vidal P</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.<br /><searchLink fieldCode="AU" term="%22Collaud+F%22">Collaud F</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.<br /><searchLink fieldCode="AU" term="%22Simon-Sola+M%22">Simon-Sola M</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.<br /><searchLink fieldCode="AU" term="%22Charles+S%22">Charles S</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.<br /><searchLink fieldCode="AU" term="%22Hardet+R%22">Hardet R</searchLink>; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.<br /><searchLink fieldCode="AU" term="%22Leborgne+C%22">Leborgne C</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.<br /><searchLink fieldCode="AU" term="%22Meliani+A%22">Meliani A</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.<br /><searchLink fieldCode="AU" term="%22Cohen-Tannoudji+M%22">Cohen-Tannoudji M</searchLink>; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.<br /><searchLink fieldCode="AU" term="%22Astord+S%22">Astord S</searchLink>; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.<br /><searchLink fieldCode="AU" term="%22Gjata+B%22">Gjata B</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.<br /><searchLink fieldCode="AU" term="%22Sellier+P%22">Sellier P</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.<br /><searchLink fieldCode="AU" term="%22van+Wittenberghe+L%22">van Wittenberghe L</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.<br /><searchLink fieldCode="AU" term="%22Vignaud+A%22">Vignaud A</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.<br /><searchLink fieldCode="AU" term="%22Boisgerault+F%22">Boisgerault F</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France.<br /><searchLink fieldCode="AU" term="%22Barkats+M%22">Barkats M</searchLink>; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.<br /><searchLink fieldCode="AU" term="%22Laforet+P%22">Laforet P</searchLink>; Paris-Est Neuromuscular Center, Pitié-Salpêtrière Hospital and Raymond Poincaré Teaching Hospital, Garches, APHP, Paris, France.<br /><searchLink fieldCode="AU" term="%22Kay+MA%22">Kay MA</searchLink>; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.<br /><searchLink fieldCode="AU" term="%22Koeberl+DD%22">Koeberl DD</searchLink>; Division of Medical Genetics, Department of Pediatrics and Department of Molecular Genetics and Microbiology, Duke University School of Medicine, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Ronzitti+G%22">Ronzitti G</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France. gronzitti@genethon.fr fmingozzi@genethon.fr.<br /><searchLink fieldCode="AU" term="%22Mingozzi+F%22">Mingozzi F</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, 91002 Evry, France. gronzitti@genethon.fr fmingozzi@genethon.fr.; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France.
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