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Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver.

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Title: Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver.
Authors: Jauze L; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France., Vie M; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France., Miagoux Q; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France., Rossiaud L; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France., Vidal P; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France., Montalvo-Romeral V; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France., Saliba H; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France., Jarrige M; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France., Polveche H; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France., Nozi J; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France., Le Brun PR; Généthon, Évry, France., Bocchialini L; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France., Francois A; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France., Cosette J; Généthon, Évry, France., Rouillon J; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France., Collaud F; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France., Bordier F; Généthon, Évry, France., Bertil-Froidevaux E; Généthon, Évry, France., Georger C; Généthon, Évry, France., van Wittenberghe L; Généthon, Évry, France., Miranda A; Généthon, Évry, France., Daniele NF; Généthon, Évry, France., Gross DA; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France., Hoch L; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France., Nissan X; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France., Ronzitti G; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.
Source: JCI insight [JCI Insight] 2024 May 16; Vol. 9 (11). Date of Electronic Publication: 2024 May 16.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 101676073 Publication Model: Electronic Cited Medium: Internet ISSN: 2379-3708 (Electronic) Linking ISSN: 23793708 NLM ISO Abbreviation: JCI Insight Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver.
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  Data: <searchLink fieldCode="AU" term="%22Jauze+L%22">Jauze L</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Vie+M%22">Vie M</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Miagoux+Q%22">Miagoux Q</searchLink>; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.<br /><searchLink fieldCode="AU" term="%22Rossiaud+L%22">Rossiaud L</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.<br /><searchLink fieldCode="AU" term="%22Vidal+P%22">Vidal P</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Montalvo-Romeral+V%22">Montalvo-Romeral V</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Saliba+H%22">Saliba H</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Jarrige+M%22">Jarrige M</searchLink>; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.<br /><searchLink fieldCode="AU" term="%22Polveche+H%22">Polveche H</searchLink>; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.<br /><searchLink fieldCode="AU" term="%22Nozi+J%22">Nozi J</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Le+Brun+PR%22">Le Brun PR</searchLink>; Généthon, Évry, France.<br /><searchLink fieldCode="AU" term="%22Bocchialini+L%22">Bocchialini L</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Francois+A%22">Francois A</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Cosette+J%22">Cosette J</searchLink>; Généthon, Évry, France.<br /><searchLink fieldCode="AU" term="%22Rouillon+J%22">Rouillon J</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Collaud+F%22">Collaud F</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Bordier+F%22">Bordier F</searchLink>; Généthon, Évry, France.<br /><searchLink fieldCode="AU" term="%22Bertil-Froidevaux+E%22">Bertil-Froidevaux E</searchLink>; Généthon, Évry, France.<br /><searchLink fieldCode="AU" term="%22Georger+C%22">Georger C</searchLink>; Généthon, Évry, France.<br /><searchLink fieldCode="AU" term="%22van+Wittenberghe+L%22">van Wittenberghe L</searchLink>; Généthon, Évry, France.<br /><searchLink fieldCode="AU" term="%22Miranda+A%22">Miranda A</searchLink>; Généthon, Évry, France.<br /><searchLink fieldCode="AU" term="%22Daniele+NF%22">Daniele NF</searchLink>; Généthon, Évry, France.<br /><searchLink fieldCode="AU" term="%22Gross+DA%22">Gross DA</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Hoch+L%22">Hoch L</searchLink>; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.<br /><searchLink fieldCode="AU" term="%22Nissan+X%22">Nissan X</searchLink>; CECS, I-STEM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.<br /><searchLink fieldCode="AU" term="%22Ronzitti+G%22">Ronzitti G</searchLink>; Généthon, Évry, France.; Université Paris-Saclay, Univ Évry, Inserm, Généthon, Integrare research unit UMR_S951, 91000 Évry, France.
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  Data: <searchLink fieldCode="JN" term="%22101676073%22">JCI insight</searchLink> [JCI Insight] 2024 May 16; Vol. 9 (11). <i>Date of Electronic Publication: </i>2024 May 16.
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