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Gene therapy using a liver-targeted AAV vector restores nucleoside and nucleotide homeostasis in a murine model of MNGIE.
Torres-Torronteras, Javier; Viscomi, Carlo; Cabrera-Pérez, Raquel; Cámara, Yolanda; Di Meo, Ivano; Barquinero, Jordi; Auricchio, Alberto; Pizzorno, Giuseppe; Hirano, Michio; Zeviani, Massimo; Martí, Ramon.
Afiliação
  • Torres-Torronteras J; 1] Mitochondrial Disorders Unit, Vall d'Hebron Institut de Recerca, Universitat Autònoma de Barcelona, Barcelona, Spain; [2] Biomedical Network Research Centre on Rare Diseases, Instituto de Salud Carlos III, Madrid, Spain;
  • Viscomi C; 1] Molecular Neurogenetics Unit, IRCCS Foundation Neurological Institute "C, Besta", Milan, Italy; [2] MRC-Mitochondrial Biology Unit, Cambridge, UK;
  • Cabrera-Pérez R; 1] Mitochondrial Disorders Unit, Vall d'Hebron Institut de Recerca, Universitat Autònoma de Barcelona, Barcelona, Spain; [2] Biomedical Network Research Centre on Rare Diseases, Instituto de Salud Carlos III, Madrid, Spain;
  • Cámara Y; 1] Mitochondrial Disorders Unit, Vall d'Hebron Institut de Recerca, Universitat Autònoma de Barcelona, Barcelona, Spain; [2] Biomedical Network Research Centre on Rare Diseases, Instituto de Salud Carlos III, Madrid, Spain;
  • Di Meo I; Molecular Neurogenetics Unit, IRCCS Foundation Neurological Institute "C, Besta", Milan, Italy;
  • Barquinero J; Gene and Cell Therapy Unit, Vall d'Hebron Institut de Recerca, Universitat Autònoma de Barcelona, Barcelona, Spain;
  • Auricchio A; Telethon Institute of Genetics and Medicine and Division of Medical Genetics, Department of Pediatrics, ''Federico II'' University, Naples, Italy;
  • Pizzorno G; Human Health and Environment Program, Desert Research Institute, Las Vegas, Nevada, USA;
  • Hirano M; Department of Neurology, Columbia University Medical Center, New York, USA.
  • Zeviani M; 1] Molecular Neurogenetics Unit, IRCCS Foundation Neurological Institute "C, Besta", Milan, Italy; [2] MRC-Mitochondrial Biology Unit, Cambridge, UK;
  • Martí R; 1] Mitochondrial Disorders Unit, Vall d'Hebron Institut de Recerca, Universitat Autònoma de Barcelona, Barcelona, Spain; [2] Biomedical Network Research Centre on Rare Diseases, Instituto de Salud Carlos III, Madrid, Spain;
Mol Ther ; 22(5): 901-7, 2014 May.
Article em En | MEDLINE | ID: mdl-24448160
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Timidina Fosforilase / Pseudo-Obstrução Intestinal / Terapia Genética / Encefalomiopatias Mitocondriais Tipo de estudo: Prognostic_studies Limite: Animals / Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2014 Tipo de documento: Article
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Timidina Fosforilase / Pseudo-Obstrução Intestinal / Terapia Genética / Encefalomiopatias Mitocondriais Tipo de estudo: Prognostic_studies Limite: Animals / Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2014 Tipo de documento: Article