AAV-based Gene Therapy Prevents Neuropathology and Results in Normal Cognitive Development in the Hyperargininemic Mouse

Complete arginase I deficiency is the least severe urea cycle disorder, characterized by hyperargininemia and infrequent episodes of hyperammonemia. Patients suffer from neurological impairment with cortical and pyramidal tract deterioration, spasticity, loss of ambulation, and seizures, and is asso...

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Autors principals: Lee, Eun K., Hu, Chuhong, Bhargava, Ragini, Ponnusamy, Ravi, Park, Hana, Novicoff, Sarah, Rozengurt, Nora, Marescau, Bart, De Deyn, Pater, Stout, David, Schlichting, Lisa, Grody, Wayne W., Cederbaum, Stephen D., Lipshutz, Gerald S.
Format: Artigo
Idioma:Inglês
Publicat: 2013
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3679314/
https://ncbi.nlm.nih.gov/pubmed/23388701
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2012.99
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