Long-term, high-level hepatic secretion of acid &#945;-glucosidase for Pompe disease achieved in non-human primates using helper-dependent adenovirus.

Rastall, D P W; Seregin, S S; Aldhamen, Y A; Kaiser, L M; Mullins, C; Liou, A; Ing, F; Pereria-Hicks, C; Godbehere-Roosa, S; Palmer, D; Ng, P; Amalfitano, A

Long-term, high-level hepatic secretion of acid α-glucosidase for Pompe disease achieved in non-human primates using helper-dependent adenovirus.

Rastall, D P W; Seregin, S S; Aldhamen, Y A; Kaiser, L M; Mullins, C; Liou, A; Ing, F; Pereria-Hicks, C; Godbehere-Roosa, S; Palmer, D; Ng, P; Amalfitano, A.

Afiliação

Rastall DP; Department of Microbiology and Molecular Genetics, College of Osteopathic Medicine, Michigan State University, East Lansing, MI, USA.
Seregin SS; Department of Microbiology and Molecular Genetics, College of Osteopathic Medicine, Michigan State University, East Lansing, MI, USA.
Aldhamen YA; Department of Microbiology and Molecular Genetics, College of Osteopathic Medicine, Michigan State University, East Lansing, MI, USA.
Kaiser LM; Department of Microbiology and Molecular Genetics, College of Osteopathic Medicine, Michigan State University, East Lansing, MI, USA.
Mullins C; Baylor College of Medicine, Houston, TX, USA.
Liou A; Baylor College of Medicine, Houston, TX, USA.
Ing F; Keck School of Medicine, Children's Hospital Los Angeles, Los Angeles, CA, USA.
Pereria-Hicks C; Department of Microbiology and Molecular Genetics, College of Osteopathic Medicine, Michigan State University, East Lansing, MI, USA.
Godbehere-Roosa S; Department of Microbiology and Molecular Genetics, College of Osteopathic Medicine, Michigan State University, East Lansing, MI, USA.
Palmer D; Baylor College of Medicine, Houston, TX, USA.
Ng P; Baylor College of Medicine, Houston, TX, USA.
Amalfitano A; Department of Microbiology and Molecular Genetics, College of Osteopathic Medicine, Michigan State University, East Lansing, MI, USA.

Gene Ther ; 23(10): 743-752, 2016 10.

Article em En | MEDLINE | ID: mdl-27367841

Assuntos

Adenoviridae/genética; Terapia Genética/métodos; Doença de Depósito de Glicogênio Tipo II/terapia; Fígado/metabolismo; alfa-Glucosidases/genética; Animais; Células Cultivadas; Diafragma/metabolismo; Terapia Genética/efeitos adversos; Vetores Genéticos/efeitos adversos; Vetores Genéticos/genética; Vírus Auxiliares/genética; Masculino; Camundongos; Camundongos Endogâmicos C57BL; Músculo Esquelético/metabolismo; Miocárdio/metabolismo; Papio; alfa-Glucosidases/metabolismo

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Doença de Depósito de Glicogênio Tipo II / Adenoviridae / Alfa-Glucosidases / Fígado Tipo de estudo: Prognostic_studies Limite: Animals Idioma: En Revista: Gene Ther Assunto da revista: GENETICA MEDICA / TERAPEUTICA Ano de publicação: 2016 Tipo de documento: Article País de afiliação: Estados Unidos

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