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Short-term Correction of Arginase Deficiency in a Neonatal Murine Model With a Helper-dependent Adenoviral Vector
Neonatal gene therapy has the potential to ameliorate abnormalities before disease onset. Our gene knockout of arginase I (AI) deficiency is characterized by increasing hyperammonemia, neurological deterioration, and early death. We constructed a helper-dependent adenoviral vector (HDV) carrying AI...
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| Main Authors: | , , , , , , , , , , |
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| Format: | Artigo |
| Language: | Inglês |
| Published: |
Nature Publishing Group
2009
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| Subjects: | |
| Online Access: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2835205/ https://ncbi.nlm.nih.gov/pubmed/19367256 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.65 |
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