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. |
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| 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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| ISSN: | 1757-4684 |
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| DOI: | 10.15252/emmm.202113968 |
