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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Détails bibliographiques
Publié dans:Mol Genet Metab
Auteurs principaux: 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.
Format: Artigo
Langue:Inglês
Publié: 2016
Sujets:
Article
Accès en ligne: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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