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Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.

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Title: Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.
Authors: Colella P; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France. Electronic address: pcolella@stanford.edu., Sellier P; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Gomez MJ; Bioinformatics Unit, CNIC, Madrid, Spain., Biferi MG; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Tanniou G; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Guerchet N; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Cohen-Tannoudji M; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Moya-Nilges M; Pasteur Institute, Paris, France., van Wittenberghe L; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Daniele N; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Gjata B; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Krijnse-Locker J; Pasteur Institute, Paris, France., Collaud F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Simon-Sola M; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Charles S; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Cagin U; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Mingozzi F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France; Spark Therapeutics, Philadelphia, PA, USA. Electronic address: federico.mingozzi@sparktx.com.
Source: EBioMedicine [EBioMedicine] 2020 Nov; Vol. 61, pp. 103052. Date of Electronic Publication: 2020 Oct 09.
Publication Type: Journal Article
Journal Info: Publisher: Elsevier B.V Country of Publication: Netherlands NLM ID: 101647039 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2352-3964 (Electronic) Linking ISSN: 23523964 NLM ISO Abbreviation: EBioMedicine Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.
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  Data: <searchLink fieldCode="AU" term="%22Colella+P%22">Colella P</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France. Electronic address: pcolella@stanford.edu.<br /><searchLink fieldCode="AU" term="%22Sellier+P%22">Sellier P</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France.<br /><searchLink fieldCode="AU" term="%22Gomez+MJ%22">Gomez MJ</searchLink>; Bioinformatics Unit, CNIC, Madrid, Spain.<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="%22Tanniou+G%22">Tanniou G</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France.<br /><searchLink fieldCode="AU" term="%22Guerchet+N%22">Guerchet N</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, 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="%22Moya-Nilges+M%22">Moya-Nilges M</searchLink>; Pasteur Institute, Paris, France.<br /><searchLink fieldCode="AU" term="%22van+Wittenberghe+L%22">van Wittenberghe L</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France.<br /><searchLink fieldCode="AU" term="%22Daniele+N%22">Daniele N</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France.<br /><searchLink fieldCode="AU" term="%22Gjata+B%22">Gjata B</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France.<br /><searchLink fieldCode="AU" term="%22Krijnse-Locker+J%22">Krijnse-Locker J</searchLink>; Pasteur Institute, Paris, France.<br /><searchLink fieldCode="AU" term="%22Collaud+F%22">Collaud F</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France.<br /><searchLink fieldCode="AU" term="%22Simon-Sola+M%22">Simon-Sola M</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France.<br /><searchLink fieldCode="AU" term="%22Charles+S%22">Charles S</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France.<br /><searchLink fieldCode="AU" term="%22Cagin+U%22">Cagin U</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France.<br /><searchLink fieldCode="AU" term="%22Mingozzi+F%22">Mingozzi F</searchLink>; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France; Spark Therapeutics, Philadelphia, PA, USA. Electronic address: federico.mingozzi@sparktx.com.
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  Data: <searchLink fieldCode="JN" term="%22101647039%22">EBioMedicine</searchLink> [EBioMedicine] 2020 Nov; Vol. 61, pp. 103052. <i>Date of Electronic Publication: </i>2020 Oct 09.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Elsevier+B%2EV%22">Elsevier B.V </searchLink><i>Country of Publication: </i>Netherlands <i>NLM ID: </i>101647039 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2352-3964 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2223523964%22">23523964 </searchLink><i>NLM ISO Abbreviation: </i>EBioMedicine <i>Subsets: </i>MEDLINE
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      – TitleFull: Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.
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              Text: 2020 Nov
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