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AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.
Wang, Lili; Bell, Peter; Morizono, Hiroki; He, Zhenning; Pumbo, Elena; Yu, Hongwei; White, John; Batshaw, Mark L; Wilson, James M.
Afiliação
  • Wang L; Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, 125 S. 31st Street, Philadelphia, PA 19104, USA.
  • Bell P; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, 125 S. 31st Street, Philadelphia, PA 19104, USA.
  • Morizono H; Center for Genetic Medicine Research, Children's Research Institute, Children's National Health System, 111 Michigan Ave., Washington, DC 20010, USA.
  • He Z; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, 125 S. 31st Street, Philadelphia, PA 19104, USA.
  • Pumbo E; Center for Genetic Medicine Research, Children's Research Institute, Children's National Health System, 111 Michigan Ave., Washington, DC 20010, USA.
  • Yu H; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, 125 S. 31st Street, Philadelphia, PA 19104, USA.
  • White J; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, 125 S. 31st Street, Philadelphia, PA 19104, USA.
  • Batshaw ML; Center for Genetic Medicine Research, Children's Research Institute, Children's National Health System, 111 Michigan Ave., Washington, DC 20010, USA.
  • Wilson JM; Gene Therapy Program, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, 125 S. 31st Street, Philadelphia, PA 19104, USA. Electronic address: wilsonjm@upenn.edu.
Mol Genet Metab ; 120(4): 299-305, 2017 04.
Article em En | MEDLINE | ID: mdl-28283349

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Ornitina Carbamoiltransferase / Envelhecimento / Doença da Deficiência de Ornitina Carbomoiltransferase / Vetores Genéticos / Cirrose Hepática Idioma: En Ano de publicação: 2017 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Ornitina Carbamoiltransferase / Envelhecimento / Doença da Deficiência de Ornitina Carbomoiltransferase / Vetores Genéticos / Cirrose Hepática Idioma: En Ano de publicação: 2017 Tipo de documento: Article