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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書誌詳細
主要な著者: 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.
フォーマット: Artigo
言語:Inglês
出版事項: The National Academy of Sciences 2002
主題:
Biological Sciences
オンライン･アクセス: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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