Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model

High fidelity animal models of human disease are essential for preclinical evaluation of novel gene and protein therapeutics. However, these studies can be complicated by exaggerated immune responses against the human transgene. Here we demonstrate that dogs with a genetic deficiency of the enzyme α...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab
Main Authors: Hinderer, Christian, Bell, Peter, Louboutin, Jean-Pierre, Katz, Nathan, Zhu, Yanqing, Lin, Gloria, Choa, Ruth, Bagel, Jessica, O'Donnell, Patricia, Fitzgerald, Caitlin A., Langan, Therese, Wang, Ping, Casal, Margret L., Haskins, Mark E., Wilson, James M.
Formato: Artigo
Idioma:Inglês
Publicado em: 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5240037/
https://ncbi.nlm.nih.gov/pubmed/27386755
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2016.06.006
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