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AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease.
Katz, Martin L; Tecedor, Luis; Chen, Yonghong; Williamson, Baye G; Lysenko, Elena; Wininger, Fred A; Young, Whitney M; Johnson, Gayle C; Whiting, Rebecca E H; Coates, Joan R; Davidson, Beverly L.
Afiliación
  • Katz ML; Department of Ophthalmology, University of Missouri, Columbia, MO 65212, USA.
  • Tecedor L; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
  • Chen Y; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
  • Williamson BG; Department of Veterinary Medicine and Surgery, College of Veterinary Medicine, University of Missouri, Columbia, MO 65211, USA.
  • Lysenko E; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
  • Wininger FA; Department of Veterinary Medicine and Surgery, College of Veterinary Medicine, University of Missouri, Columbia, MO 65211, USA.
  • Young WM; Department of Ophthalmology, University of Missouri, Columbia, MO 65212, USA.
  • Johnson GC; Department of Veterinary Pathobiology, College of Veterinary Medicine, University of Missouri, Columbia, MO 65211, USA.
  • Whiting RE; Department of Veterinary Medicine and Surgery, College of Veterinary Medicine, University of Missouri, Columbia, MO 65211, USA.
  • Coates JR; Department of Veterinary Medicine and Surgery, College of Veterinary Medicine, University of Missouri, Columbia, MO 65211, USA.
  • Davidson BL; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA. davidsonbl@email.chop.edu.
Sci Transl Med ; 7(313): 313ra180, 2015 Nov 11.
Article en En | MEDLINE | ID: mdl-26560358

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Transducción Genética / Dipeptidil-Peptidasas y Tripeptidil-Peptidasas / Serina Proteasas / Terapia de Reemplazo Enzimático / Aminopeptidasas / Lipofuscinosis Ceroideas Neuronales Tipo de estudio: Prognostic_studies Límite: Animals Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2015 Tipo del documento: Article País de afiliación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Transducción Genética / Dipeptidil-Peptidasas y Tripeptidil-Peptidasas / Serina Proteasas / Terapia de Reemplazo Enzimático / Aminopeptidasas / Lipofuscinosis Ceroideas Neuronales Tipo de estudio: Prognostic_studies Límite: Animals Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2015 Tipo del documento: Article País de afiliación: Estados Unidos
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