Intravitreal enzyme replacement inhibits progression of retinal degeneration in canine CLN2 neuronal ceroid lipofuscinosis.

Whiting, Rebecca E H; Robinson Kick, Grace; Ota-Kuroki, Juri; Lim, Stefanie; Castaner, Leilani J; Jensen, Cheryl A; Kowal, Joseph; Nguyen, Annalisa; Corado, Carley; O'Neill, Charles A; Katz, Martin L

Whiting, Rebecca E H; Robinson Kick, Grace; Ota-Kuroki, Juri; Lim, Stefanie; Castaner, Leilani J; Jensen, Cheryl A; Kowal, Joseph; Nguyen, Annalisa; Corado, Carley; O'Neill, Charles A; Katz, Martin L.

Afiliação

Whiting REH; Neurodegenerative Diseases Research Laboratory, University of Missouri, Columbia, MO, 65212, USA.
Robinson Kick G; Neurodegenerative Diseases Research Laboratory, University of Missouri, Columbia, MO, 65212, USA.
Ota-Kuroki J; Neurodegenerative Diseases Research Laboratory, University of Missouri, Columbia, MO, 65212, USA.
Lim S; Neurodegenerative Diseases Research Laboratory, University of Missouri, Columbia, MO, 65212, USA.
Castaner LJ; Neurodegenerative Diseases Research Laboratory, University of Missouri, Columbia, MO, 65212, USA.
Jensen CA; Neurodegenerative Diseases Research Laboratory, University of Missouri, Columbia, MO, 65212, USA.
Kowal J; Neurodegenerative Diseases Research Laboratory, University of Missouri, Columbia, MO, 65212, USA.
Nguyen A; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
Corado C; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
O'Neill CA; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
Katz ML; Neurodegenerative Diseases Research Laboratory, University of Missouri, Columbia, MO, 65212, USA. Electronic address: katzm@health.missouri.edu.

Exp Eye Res ; 198: 108135, 2020 09.

Article em En | MEDLINE | ID: mdl-32634395

Assuntos

Aminopeptidases/administração & dosagem; Dipeptidil Peptidases e Tripeptidil Peptidases/administração & dosagem; Terapia de Reposição de Enzimas/métodos; Lipofuscinoses Ceroides Neuronais/complicações; Serina Proteases/administração & dosagem; Animais; Modelos Animais de Doenças; Progressão da Doença; Cães; Eletrorretinografia; Injeções Intravítreas; Lipofuscinoses Ceroides Neuronais/diagnóstico; Lipofuscinoses Ceroides Neuronais/tratamento farmacológico; Retina/metabolismo; Retina/patologia; Degeneração Retiniana/patologia; Tripeptidil-Peptidase 1

Palavras-chave

Enzyme replacement therapy; Genetic diseases; Intravitreal drug delivery; Lysosomal storage disease; Neuronal ceroid lipofuscinosis; Retinal degeneration

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Dipeptidil Peptidases e Tripeptidil Peptidases / Serina Proteases / Terapia de Reposição de Enzimas / Aminopeptidases / Lipofuscinoses Ceroides Neuronais Idioma: En Ano de publicação: 2020 Tipo de documento: Article

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