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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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Main Authors: | , , , , , , , , |
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格式: | Artigo |
語言: | Inglês |
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Mary Ann Liebert, Inc.
2012
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在線閱讀: | 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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