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Efficacy of adeno-associated virus gene therapy in a MNGIE murine model enhanced by chronic exposure to nucleosides.
Vila-Julià, Ferran; Cabrera-Pérez, Raquel; Cámara, Yolanda; Molina-Berenguer, Miguel; Lope-Piedrafita, Silvia; Hirano, Michio; Mingozzi, Federico; Torres-Torronteras, Javier; Martí, Ramon.
Affiliation
  • Vila-Julià F; Research Group on Neuromuscular and Mitochondrial Diseases, Vall d'Hebron Research Institute, Universitat Autònoma de Barcelona, and Biomedical Network Research Centre on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Barcelona, Catalonia, Spain.
  • Cabrera-Pérez R; Research Group on Neuromuscular and Mitochondrial Diseases, Vall d'Hebron Research Institute, Universitat Autònoma de Barcelona, and Biomedical Network Research Centre on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Barcelona, Catalonia, Spain.
  • Cámara Y; Research Group on Neuromuscular and Mitochondrial Diseases, Vall d'Hebron Research Institute, Universitat Autònoma de Barcelona, and Biomedical Network Research Centre on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Barcelona, Catalonia, Spain.
  • Molina-Berenguer M; Research Group on Neuromuscular and Mitochondrial Diseases, Vall d'Hebron Research Institute, Universitat Autònoma de Barcelona, and Biomedical Network Research Centre on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Barcelona, Catalonia, Spain.
  • Lope-Piedrafita S; Servei de Ressonància Magnètica Nuclear, Universitat Autònoma de Barcelona, Cerdanyola del Vallès, Catalonia, Spain; Centro de Investigación Biomédica en Red en Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), Cerdanyola del Vallès, Catalonia, Spain.
  • Hirano M; Department of Neurology, H. Houston Merritt Neuromuscular Research Center, Columbia University Irving Medical Center, New York, NY, United States.
  • Mingozzi F; Spark Therapeutics, Philadelphia, PA 19104, United States.
  • Torres-Torronteras J; Research Group on Neuromuscular and Mitochondrial Diseases, Vall d'Hebron Research Institute, Universitat Autònoma de Barcelona, and Biomedical Network Research Centre on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Barcelona, Catalonia, Spain. Electronic address: javier.torres@vhir.org.
  • Martí R; Research Group on Neuromuscular and Mitochondrial Diseases, Vall d'Hebron Research Institute, Universitat Autònoma de Barcelona, and Biomedical Network Research Centre on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Barcelona, Catalonia, Spain. Electronic address: ramon.marti@vhir.org.
EBioMedicine ; 62: 103133, 2020 Dec.
Article in En | MEDLINE | ID: mdl-33232869
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Intestinal Pseudo-Obstruction / Genetic Therapy / Ophthalmoplegia / Dependovirus / Muscular Dystrophy, Oculopharyngeal / Genetic Vectors / Nucleosides Type of study: Prognostic_studies / Risk_factors_studies Limits: Animals / Humans Language: En Journal: EBioMedicine Year: 2020 Document type: Article Affiliation country: España Country of publication: Países Bajos
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Intestinal Pseudo-Obstruction / Genetic Therapy / Ophthalmoplegia / Dependovirus / Muscular Dystrophy, Oculopharyngeal / Genetic Vectors / Nucleosides Type of study: Prognostic_studies / Risk_factors_studies Limits: Animals / Humans Language: En Journal: EBioMedicine Year: 2020 Document type: Article Affiliation country: España Country of publication: Países Bajos