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

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.

Afiliação

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 em En | MEDLINE | ID: mdl-26560358

Assuntos

Aminopeptidases/genética; Dipeptidil Peptidases e Tripeptidil Peptidases/genética; Terapia de Reposição de Enzimas; Lipofuscinoses Ceroides Neuronais/genética; Lipofuscinoses Ceroides Neuronais/terapia; Serina Proteases/genética; Transdução Genética; Aminopeptidases/líquido cefalorraquidiano; Aminopeptidases/deficiência; Animais; Ventrículos Cerebrais/metabolismo; Dependovirus/genética; Dipeptidil Peptidases e Tripeptidil Peptidases/líquido cefalorraquidiano; Dipeptidil Peptidases e Tripeptidil Peptidases/deficiência; Modelos Animais de Doenças; Cães; Vetores Genéticos/administração & dosagem; Serina Proteases/líquido cefalorraquidiano; Serina Proteases/deficiência; Tripeptidil-Peptidase 1

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transdução Genética / Dipeptidil Peptidases e Tripeptidil Peptidases / Serina Proteases / Terapia de Reposição de Enzimas / Aminopeptidases / Lipofuscinoses Ceroides Neuronais Tipo de estudo: Prognostic_studies Limite: Animals Idioma: En Ano de publicação: 2015 Tipo de documento: Article

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