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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...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2014
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Materias: | |
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 |