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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Autors principals: Gau, Chia-Ling, Rosenblatt, Robin A, Cerullo, Vincenzo, Lay, Fides D, Dow, Adrienne C, Livesay, Justin, Brunetti-Pierri, Nicola, Lee, Brendan, Cederbaum, Stephen D, Grody, Wayne W, Lipshutz, Gerald S
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2009
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Original Articles
Accés en línia: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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