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...
Guardat en:
| Publicat a: | Proc Natl Acad Sci U S A |
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| Autors principals: | , , , , , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
National Academy of Sciences
1999
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| Matèries: | |
| Accés en línia: | 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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