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Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicity.

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Title: Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicity.
Authors: Eggers M; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Vannoy CH; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Huang J; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Purushothaman P; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Brassard J; Jacqueline Brassard Toxicologic Pathology Consulting, Tustin, CA, USA., Fonck C; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Meng H; Department of Pathology and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI, USA., Prom MJ; Department of Pathology and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI, USA., Lawlor MW; Department of Pathology and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI, USA., Cunningham J; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Sadhu C; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Mavilio F; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA.; Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy.
Source: EMBO molecular medicine [EMBO Mol Med] 2022 Jan 11; Vol. 14 (1), pp. e13968. Date of Electronic Publication: 2021 Dec 01.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: EMBO Press Country of Publication: Germany NLM ID: 101487380 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1757-4684 (Electronic) Linking ISSN: 17574676 NLM ISO Abbreviation: EMBO Mol Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicity.
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  Data: <searchLink fieldCode="AU" term="%22Eggers+M%22">Eggers M</searchLink>; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Vannoy+CH%22">Vannoy CH</searchLink>; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Huang+J%22">Huang J</searchLink>; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Purushothaman+P%22">Purushothaman P</searchLink>; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Brassard+J%22">Brassard J</searchLink>; Jacqueline Brassard Toxicologic Pathology Consulting, Tustin, CA, USA.<br /><searchLink fieldCode="AU" term="%22Fonck+C%22">Fonck C</searchLink>; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Meng+H%22">Meng H</searchLink>; Department of Pathology and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI, USA.<br /><searchLink fieldCode="AU" term="%22Prom+MJ%22">Prom MJ</searchLink>; Department of Pathology and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI, USA.<br /><searchLink fieldCode="AU" term="%22Lawlor+MW%22">Lawlor MW</searchLink>; Department of Pathology and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI, USA.<br /><searchLink fieldCode="AU" term="%22Cunningham+J%22">Cunningham J</searchLink>; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Sadhu+C%22">Sadhu C</searchLink>; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Mavilio+F%22">Mavilio F</searchLink>; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA.; Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy.
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  Data: <searchLink fieldCode="JN" term="%22101487380%22">EMBO molecular medicine</searchLink> [EMBO Mol Med] 2022 Jan 11; Vol. 14 (1), pp. e13968. <i>Date of Electronic Publication: </i>2021 Dec 01.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22EMBO+Press%22">EMBO Press </searchLink><i>Country of Publication: </i>Germany <i>NLM ID: </i>101487380 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1757-4684 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2217574676%22">17574676 </searchLink><i>NLM ISO Abbreviation: </i>EMBO Mol Med <i>Subsets: </i>MEDLINE
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