Intravitreal gene therapy protects against retinal dysfunction and degeneration in sheep with CLN5 Batten disease.

Murray, Samantha J; Russell, Katharina N; Melzer, Tracy R; Gray, Steven J; Heap, Stephen J; Palmer, David N; Mitchell, Nadia L

Murray, Samantha J; Russell, Katharina N; Melzer, Tracy R; Gray, Steven J; Heap, Stephen J; Palmer, David N; Mitchell, Nadia L.

Afiliação

Murray SJ; Faculty of Agricultural and Life Sciences, Lincoln University, Lincoln, 7647, New Zealand.
Russell KN; Faculty of Agricultural and Life Sciences, Lincoln University, Lincoln, 7647, New Zealand.
Melzer TR; Department of Medicine, University of Otago, Christchurch and the New Zealand Brain Research Institute, Christchurch, 8011, New Zealand.
Gray SJ; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, 75390, USA.
Heap SJ; McMaster & Heap Veterinary Practice, Christchurch, 8025, New Zealand.
Palmer DN; Faculty of Agricultural and Life Sciences, Lincoln University, Lincoln, 7647, New Zealand; Department of Radiology, University of Otago, Christchurch, 8140, New Zealand.
Mitchell NL; Faculty of Agricultural and Life Sciences, Lincoln University, Lincoln, 7647, New Zealand; Department of Radiology, University of Otago, Christchurch, 8140, New Zealand. Electronic address: Nadia.Mitchell@lincoln.ac.nz.

Exp Eye Res ; 207: 108600, 2021 06.

Article em En | MEDLINE | ID: mdl-33930398

Assuntos

Terapia Genética/métodos; Proteínas de Membrana Lisossomal/genética; Lipofuscinoses Ceroides Neuronais/terapia; Degeneração Retiniana/terapia; Animais; Dependovirus/genética; Modelos Animais de Doenças; Eletrorretinografia; Feminino; Vetores Genéticos; Proteína Glial Fibrilar Ácida/metabolismo; Injeções Intravítreas; Proteína 1 de Membrana Associada ao Lisossomo/metabolismo; Lipofuscinoses Ceroides Neuronais/metabolismo; Lipofuscinoses Ceroides Neuronais/fisiopatologia; Retina/metabolismo; Retina/fisiopatologia; Degeneração Retiniana/metabolismo; Degeneração Retiniana/fisiopatologia; Ovinos

Palavras-chave

AAV9; CLN5; CLN6; Electroretinography; Gene therapy; Intravitreal; Neuronal ceroid lipofuscinosis; Sheep

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Degeneração Retiniana / Terapia Genética / Proteínas de Membrana Lisossomal / Lipofuscinoses Ceroides Neuronais Limite: Animals Idioma: En Ano de publicação: 2021 Tipo de documento: Article

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