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Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease

BACKGROUND: Lysosomal storage disorders such as Pompe disease can be more effectively treated, if immune tolerance to enzyme or gene replacement therapy can be achieved. Alternatively, immune responses against acid α-glucosidase (GAA) might be evaded in Pompe disease through muscle-specific expressi...

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מידע ביבליוגרפי
Main Authors: Sun, Baodong, Li, Songtao, Bird, Andrew, Yi, Haiqing, Kemper, Alex, Koeberl, Dwight D.
פורמט: Artigo
שפה:Inglês
יצא לאור: 2010
נושאים:
גישה מקוונת:https://ncbi.nlm.nih.gov/pmc/articles/PMC3655728/
https://ncbi.nlm.nih.gov/pubmed/20967919
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jgm.1511
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