Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease

Pompe disease is a progressive neuromuscular disorder caused by lysosomal accumulation of glycogen from a deficiency in acid alpha-glucosidase (GAA). Replacement of the missing enzyme is available by repeated protein infusions; however, efficacy is limited by immune response and inability to restore...

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Bibliografische gegevens
Gepubliceerd in:Hum Gene Ther
Hoofdauteurs: Doerfler, Phillip A., Todd, Adrian G., Clément, Nathalie, Falk, Darin J., Nayak, Sushrusha, Herzog, Roland W., Byrne, Barry J.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Mary Ann Liebert, Inc. 2016
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Research Articles
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4741206/
https://ncbi.nlm.nih.gov/pubmed/26603344
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.103
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