Antisense oligonucleotide treatment rescues UBE3A expression and multiple phenotypes of an Angelman syndrome mouse model.

Milazzo, Claudia; Mientjes, Edwin J; Wallaard, Ilse; Rasmussen, Søren Vestergaard; Erichsen, Kamille Dumong; Kakunuri, Tejaswini; van der Sman, A S Elise; Kremer, Thomas; Miller, Meghan T; Hoener, Marius C; Elgersma, Ype

Milazzo, Claudia; Mientjes, Edwin J; Wallaard, Ilse; Rasmussen, Søren Vestergaard; Erichsen, Kamille Dumong; Kakunuri, Tejaswini; van der Sman, A S Elise; Kremer, Thomas; Miller, Meghan T; Hoener, Marius C; Elgersma, Ype.

Afiliação

Milazzo C; Departments of Clinical Genetics and Neuroscience and.
Mientjes EJ; ENCORE Expertise Center for Neurodevelopmental Disorders, Erasmus MC, Rotterdam, Netherlands.
Wallaard I; Departments of Clinical Genetics and Neuroscience and.
Rasmussen SV; ENCORE Expertise Center for Neurodevelopmental Disorders, Erasmus MC, Rotterdam, Netherlands.
Erichsen KD; Departments of Clinical Genetics and Neuroscience and.
Kakunuri T; ENCORE Expertise Center for Neurodevelopmental Disorders, Erasmus MC, Rotterdam, Netherlands.
van der Sman ASE; Therapeutic Modalities, Roche Innovation Center Copenhagen, F. Hoffmann-La Roche Ltd., Horsholm, Denmark.
Kremer T; Therapeutic Modalities, Roche Innovation Center Copenhagen, F. Hoffmann-La Roche Ltd., Horsholm, Denmark.
Miller MT; Departments of Clinical Genetics and Neuroscience and.
Hoener MC; ENCORE Expertise Center for Neurodevelopmental Disorders, Erasmus MC, Rotterdam, Netherlands.
Elgersma Y; Departments of Clinical Genetics and Neuroscience and.

JCI Insight ; 6(15)2021 08 09.

Article em En | MEDLINE | ID: mdl-34369389

Assuntos

Síndrome de Angelman; Oligonucleotídeos Antissenso/uso terapêutico; Ubiquitina-Proteína Ligases/metabolismo; Síndrome de Angelman/genética; Síndrome de Angelman/metabolismo; Animais; Variação Biológica da População; Modelos Animais de Doenças; Perfilação da Expressão Gênica; Inativação Gênica; Camundongos; Reparo Gênico Alvo-Dirigido/métodos; Resultado do Tratamento

Palavras-chave

Development; Mouse models; Neurodevelopment; Neurological disorders; Neuroscience

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Oligonucleotídeos Antissenso / Síndrome de Angelman / Ubiquitina-Proteína Ligases Tipo de estudo: Prognostic_studies Limite: Animals Idioma: En Revista: JCI Insight Ano de publicação: 2021 Tipo de documento: Article

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