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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2013
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3679314/
https://www.ncbi.nlm.nih.gov/pubmed/23388701
http://dx.doi.org/10.1038/gt.2012.99

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3679314/
https://www.ncbi.nlm.nih.gov/pubmed/23388701
http://dx.doi.org/10.1038/gt.2012.99

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