Long-term Survival of the Juvenile Lethal Arginase-deficient Mouse With AAV Gene Therapy

Arginase deficiency is characterized by hyperargininemia and infrequent episodes of hyperammonemia. Human patients suffer from neurological impairment with spasticity, loss of ambulation, seizures, and severe mental and growth retardation. In a murine model, onset of the phenotypic abnormality is he...

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Bibliographische Detailangaben
Hauptverfasser: Lee, Eun K., Hu, Chuhong, Bhargava, Ragini, Rozengurt, Nora, Stout, David, Grody, Wayne W, Cederbaum, Stephen D, Lipshutz, Gerald S
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2012
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3464644/
https://ncbi.nlm.nih.gov/pubmed/22760543
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.129
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