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Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy.
Yoon, Sea Young; Hunter, Jacqueline E; Chawla, Sanjeev; Clarke, Dana L; Molony, Caitlyn; O'Donnell, Patricia A; Bagel, Jessica H; Kumar, Manoj; Poptani, Harish; Vite, Charles H; Wolfe, John H.
Afiliación
  • Yoon SY; Research Institute of Children's Hospital of Philadelphia, Philadelphia, USA.
  • Hunter JE; Research Institute of Children's Hospital of Philadelphia, Philadelphia, USA.
  • Chawla S; Department of Radiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, USA.
  • Clarke DL; W.F. Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, USA.
  • Molony C; W.F. Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, USA.
  • O'Donnell PA; W.F. Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, USA.
  • Bagel JH; W.F. Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, USA.
  • Kumar M; Department of Radiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, USA.
  • Poptani H; Department of Radiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, USA.
  • Vite CH; W.F. Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, USA.
  • Wolfe JH; Research Institute of Children's Hospital of Philadelphia, Philadelphia, USA.
Brain ; 143(7): 2058-2072, 2020 07 01.
Article en En | MEDLINE | ID: mdl-32671406
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Encéfalo / Terapia Genética / Dependovirus / Modelos Animales de Enfermedad / Vectores Genéticos / Alfa-Manosidosis Límite: Animals Idioma: En Revista: Brain Año: 2020 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Encéfalo / Terapia Genética / Dependovirus / Modelos Animales de Enfermedad / Vectores Genéticos / Alfa-Manosidosis Límite: Animals Idioma: En Revista: Brain Año: 2020 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido