Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction.

Podetz-Pedersen, Kelly M; Laoharawee, Kanut; Singh, Sajya; Nguyen, Tam T; Smith, Miles C; Temme, Alexa; Kozarsky, Karen; McIvor, R Scott; Belur, Lalitha R

Podetz-Pedersen, Kelly M; Laoharawee, Kanut; Singh, Sajya; Nguyen, Tam T; Smith, Miles C; Temme, Alexa; Kozarsky, Karen; McIvor, R Scott; Belur, Lalitha R.

Afiliação

Podetz-Pedersen KM; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.
Laoharawee K; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.
Singh S; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.
Nguyen TT; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.
Smith MC; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.
Temme A; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.
Kozarsky K; REGENXBIO, Inc., Rockville, Maryland, USA.
McIvor RS; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.
Belur LR; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.

Hum Gene Ther ; 34(1-2): 8-18, 2023 01.

Article em En | MEDLINE | ID: mdl-36541357

Assuntos

Disfunção Cognitiva; Iduronato Sulfatase; Mucopolissacaridose II; Mucopolissacaridose I; Doenças do Sistema Nervoso; Humanos; Animais; Camundongos; Idoso; Qualidade de Vida; Mucopolissacaridose II/genética; Mucopolissacaridose II/terapia; Mucopolissacaridose I/genética; Mucopolissacaridose I/terapia; Sistema Nervoso Central/metabolismo; Iduronidase/genética; Iduronidase/metabolismo; Iduronato Sulfatase/genética; Disfunção Cognitiva/metabolismo; Glicosaminoglicanos/metabolismo; Modelos Animais de Doenças

Palavras-chave

AAV; MPS I; MPS II; gene therapy; neurodevelopment

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Mucopolissacaridose II / Mucopolissacaridose I / Disfunção Cognitiva / Iduronato Sulfatase / Doenças do Sistema Nervoso Tipo de estudo: Prognostic_studies Limite: Aged / Animals / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article

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