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Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-α-glucosidase

This report demonstrates that a single intravenous administration of a gene therapy vector can potentially result in the correction of all affected muscles in a mouse model of a human genetic muscle disease. These results were achieved by capitalizing both on the positive attributes of modified aden...

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Udgivet i:Proc Natl Acad Sci U S A
Principais autores: Amalfitano, A., McVie-Wylie, A. J., Hu, H., Dawson, T. L., Raben, N., Plotz, P., Chen, Y. T.
Format: Artigo
Sprog:Inglês
Udgivet: National Academy of Sciences 1999
Fag:
Biological Sciences
Online adgang:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC17698/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/10430861/
https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.96.16.8861
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