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A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease.

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Title: A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease.
Authors: Sellier P; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France., Collaud F; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France., Benchekroun YK; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France., Jimenez V; Center of Animal Biotechnology and Gene Therapy (CBATEG), Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Department of Biochemistry and Molecular Biology, School of Veterinary Medicine, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain., Leon X; Center of Animal Biotechnology and Gene Therapy (CBATEG), Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Department of Biochemistry and Molecular Biology, School of Veterinary Medicine, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain., Daniele N; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France., Pham QH; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel (VUB), 1090 Brussels, Belgium., El Andari J; Department of Infectious Diseases/Virology, Section Viral Vector Technologies, BioQuant Center, Medical Faculty, University of Heidelberg, 69120 Heidelberg, Germany; German Center for Infection Research (DZIF) and German Center for Cardiovascular Research (DZHK), Partner Site Heidelberg, Heidelberg, Germany., VandenDriessche T; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel (VUB), 1090 Brussels, Belgium., Chuah MK; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel (VUB), 1090 Brussels, Belgium.; Department of Cardiovascular Sciences, Center for Molecular & Vascular Biology, University of Leuven, 3000 Leuven, Belgium., Grimm D; Department of Infectious Diseases/Virology, Section Viral Vector Technologies, BioQuant Center, Medical Faculty, University of Heidelberg, 69120 Heidelberg, Germany; German Center for Infection Research (DZIF) and German Center for Cardiovascular Research (DZHK), Partner Site Heidelberg, Heidelberg, Germany., Bosch F; Center of Animal Biotechnology and Gene Therapy (CBATEG), Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Department of Biochemistry and Molecular Biology, School of Veterinary Medicine, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain., Mingozzi F; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France., Ronzitti G; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2025 Apr 09; Vol. 33 (2), pp. 101464. Date of Electronic Publication: 2025 Apr 09 (Print Publication: 2025).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: In Process
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  Data: A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease.
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  Data: <searchLink fieldCode="AU" term="%22Sellier+P%22">Sellier P</searchLink>; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Collaud+F%22">Collaud F</searchLink>; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Benchekroun+YK%22">Benchekroun YK</searchLink>; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Jimenez+V%22">Jimenez V</searchLink>; Center of Animal Biotechnology and Gene Therapy (CBATEG), Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Department of Biochemistry and Molecular Biology, School of Veterinary Medicine, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain.<br /><searchLink fieldCode="AU" term="%22Leon+X%22">Leon X</searchLink>; Center of Animal Biotechnology and Gene Therapy (CBATEG), Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Department of Biochemistry and Molecular Biology, School of Veterinary Medicine, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain.<br /><searchLink fieldCode="AU" term="%22Daniele+N%22">Daniele N</searchLink>; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Pham+QH%22">Pham QH</searchLink>; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel (VUB), 1090 Brussels, Belgium.<br /><searchLink fieldCode="AU" term="%22El+Andari+J%22">El Andari J</searchLink>; Department of Infectious Diseases/Virology, Section Viral Vector Technologies, BioQuant Center, Medical Faculty, University of Heidelberg, 69120 Heidelberg, Germany; German Center for Infection Research (DZIF) and German Center for Cardiovascular Research (DZHK), Partner Site Heidelberg, Heidelberg, Germany.<br /><searchLink fieldCode="AU" term="%22VandenDriessche+T%22">VandenDriessche T</searchLink>; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel (VUB), 1090 Brussels, Belgium.<br /><searchLink fieldCode="AU" term="%22Chuah+MK%22">Chuah MK</searchLink>; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel (VUB), 1090 Brussels, Belgium.; Department of Cardiovascular Sciences, Center for Molecular & Vascular Biology, University of Leuven, 3000 Leuven, Belgium.<br /><searchLink fieldCode="AU" term="%22Grimm+D%22">Grimm D</searchLink>; Department of Infectious Diseases/Virology, Section Viral Vector Technologies, BioQuant Center, Medical Faculty, University of Heidelberg, 69120 Heidelberg, Germany; German Center for Infection Research (DZIF) and German Center for Cardiovascular Research (DZHK), Partner Site Heidelberg, Heidelberg, Germany.<br /><searchLink fieldCode="AU" term="%22Bosch+F%22">Bosch F</searchLink>; Center of Animal Biotechnology and Gene Therapy (CBATEG), Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Department of Biochemistry and Molecular Biology, School of Veterinary Medicine, Universitat Autònoma de Barcelona, 08193 Bellaterra, Spain; Centro de Investigación Biomédica en Red de Diabetes y Enfermedades Metabólicas Asociadas (CIBERDEM), 28029 Madrid, Spain.<br /><searchLink fieldCode="AU" term="%22Mingozzi+F%22">Mingozzi F</searchLink>; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Ronzitti+G%22">Ronzitti G</searchLink>; Université Paris-Saclay, University Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Genethon, 91000 Evry, France.
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