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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Bibliografski detalji
Glavni autori: Lee, Eun K., Hu, Chuhong, Bhargava, Ragini, Rozengurt, Nora, Stout, David, Grody, Wayne W, Cederbaum, Stephen D, Lipshutz, Gerald S
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2012
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Original Article
Online pristup: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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