Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

Dogs with mucopolysaccharidosis VII (MPS VII) were injected intravenously at 2–3 days of age with a retroviral vector (RV) expressing canine β-glucuronidase (cGUSB). Five animals received RV alone, and two dogs received hepatocyte growth factor (HGF) before RV in an attempt to increase transduction...

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Detalhes bibliográficos
Main Authors: Ponder, Katherine Parker, Melniczek, John R., Xu, Lingfei, Weil, Margaret A., O'Malley, Thomas M., O'Donnell, Patricia A., Knox, Van W., Aguirre, Gustavo D., Mazrier, Hamutal, Ellinwood, N. Matthew, Sleeper, Meg, Maguire, Albert M., Volk, Susan W., Mango, Robert L., Zweigle, Jean, Wolfe, John H., Haskins, Mark E.
Formato: Artigo
Idioma:Inglês
Publicado em: The National Academy of Sciences 2002
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC130593/
https://ncbi.nlm.nih.gov/pubmed/12232044
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.192353499
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