Immunodominant Liver-Specific Expression Suppresses Transgene-Directed Immune Responses in Murine Pompe Disease

Pompe disease can be treated effectively, if immune tolerance to enzyme replacement therapy (ERT) with acid α-glucosidase (GAA) is present. An adeno-associated viral (AAV) vector carrying a liver-specific regulatory cassette to drive GAA expression (AAV-LSPhGAA) established immune tolerance in GAA k...

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Détails bibliographiques
Auteurs principaux: Zhang, Ping, Sun, Baodong, Osada, Takuya, Rodriguiz, Ramona, Yang, Xiao Yi, Luo, Xiaoyan, Kemper, Alex R., Clay, Timothy M., Koeberl, Dwight D.
Format: Artigo
Langue:Inglês
Publié: Mary Ann Liebert, Inc. 2012
Sujets:
Research Articles
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3360500/
https://ncbi.nlm.nih.gov/pubmed/22260439
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.063
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