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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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.
Format: Artigo
Jezik:Inglês
Izdano: The National Academy of Sciences 2002
Teme:
Biological Sciences
Online dostop: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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