Long-term correction of biochemical and neurological abnormalities in MLD mice model by neonatal systemic injection of an AAV serotype 9 vector.

Miyake, N; Miyake, K; Asakawa, N; Yamamoto, M; Shimada, T

Miyake, N; Miyake, K; Asakawa, N; Yamamoto, M; Shimada, T.

Afiliação

Miyake N; Division of Gene Therapy Research, Department of Biochemistry and Molecular Biology, Center for Advanced Medical Technology, Nippon Medical School, Tokyo, Japan.
Miyake K; Division of Gene Therapy Research, Department of Biochemistry and Molecular Biology, Center for Advanced Medical Technology, Nippon Medical School, Tokyo, Japan.
Asakawa N; Division of Gene Therapy Research, Department of Biochemistry and Molecular Biology, Center for Advanced Medical Technology, Nippon Medical School, Tokyo, Japan.
Yamamoto M; Division of Gene Therapy Research, Department of Biochemistry and Molecular Biology, Center for Advanced Medical Technology, Nippon Medical School, Tokyo, Japan.
Shimada T; Division of Gene Therapy Research, Department of Biochemistry and Molecular Biology, Center for Advanced Medical Technology, Nippon Medical School, Tokyo, Japan.

Gene Ther ; 21(4): 427-33, 2014 Apr.

Article em En | MEDLINE | ID: mdl-24572788

Assuntos

Dependovirus/genética; Terapia Genética; Leucodistrofia Metacromática/genética; Leucodistrofia Metacromática/terapia; Animais; Barreira Hematoencefálica; Cerebrosídeo Sulfatase/genética; Cerebrosídeo Sulfatase/uso terapêutico; Modelos Animais de Doenças; Vetores Genéticos; Proteínas de Fluorescência Verde/genética; Humanos; Leucodistrofia Metacromática/patologia; Camundongos

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Leucodistrofia Metacromática Limite: Animals / Humans Idioma: En Ano de publicação: 2014 Tipo de documento: Article

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