Advancements in AAV-mediated Gene Therapy for Pompe Disease.

Salabarria, S M; Nair, J; Clement, N; Smith, B K; Raben, N; Fuller, D D; Byrne, B J; Corti, M

Salabarria, S M; Nair, J; Clement, N; Smith, B K; Raben, N; Fuller, D D; Byrne, B J; Corti, M.

Afiliação

Salabarria SM; Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, Floria, USA.
Nair J; Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, Floria, USA.
Clement N; Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, Floria, USA.
Smith BK; Department of Physical Therapy and Center for Respiratory Research and Rehabilitation, University of Florida, Gainesville, Florida, USA.
Raben N; Laboratory of Protein Trafficking and Organelle Biology, Cell and Developmental Biology Center, National Heart, Lung and Blood Institute, NIH, Bethesda, Maryland, USA.
Fuller DD; Department of Physical Therapy and Center for Respiratory Research and Rehabilitation, University of Florida, Gainesville, Florida, USA.
Byrne BJ; Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, Floria, USA.
Corti M; Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, Floria, USA.

J Neuromuscul Dis ; 7(1): 15-31, 2020.

Article em En | MEDLINE | ID: mdl-31796685

Assuntos

Dependovirus; Terapia Genética; Doença de Depósito de Glicogênio Tipo II/terapia; Animais; Humanos

Palavras-chave

Pompe disease; adeno-associated virus (AAV); autophagy; immune-response; intramuscular; intrathecal; intravenous; lysosomes; manufacturing; promoter

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Doença de Depósito de Glicogênio Tipo II / Dependovirus Idioma: En Ano de publicação: 2020 Tipo de documento: Article

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