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Minimal Ureagenesis is Necessary for Survival in the Murine Model of Hyperargininemia Treated by AAV-based Gene Therapy

Hyperammonemia is less severe in arginase 1 deficiency compared with other urea cycle defects. Affected patients manifest hyperargininemia and infrequent episodes of hyperammonemia. Patients typically suffer from neurological impairment with cortical and pyramidal tract deterioration, spasticity, lo...

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Detalles Bibliográficos
Autores principales:	Hu, Chuhong, Tai, Denise S., Park, Hana, Cantero-Nieto, Gloria, Chan, Emily, Yudkoff, Marc, Cederbaum, Stephen D., Lipshutz, Gerald S.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2014
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4320015/ https://www.ncbi.nlm.nih.gov/pubmed/25474440 http://dx.doi.org/10.1038/gt.2014.106

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