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N-CARBAMYLGLUTAMATE ENHANCEMENT OF UREAGENESIS LEADS TO DISCOVERY OF A NOVEL DELETERIOUS MUTATION IN A NEWLY DEFINED ENHANCER OF THE NAGS GENE AND TO EFFECTIVE THERAPY

NAGS catalyzes the conversion of glutamate and acetyl-CoA to N-acetylglutamate (NAG) the essential allosteric activator of carbamyl phosphate synthetase I, the first urea cycle enzyme in mammals. A 17-year-old female with recurrent hyperammonemia attacks, the cause of which remained undiagnosed for...

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Detalhes bibliográficos
Main Authors: Heibel, Sandra K., Mew, Nicholas Ah, Caldovic, Ljubica, Daikhin, Yevgeny, Yudkoff, Marc, Tuchman, Mendel
Formato: Artigo
Idioma:Inglês
Publicado em: 2011
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3976964/
https://ncbi.nlm.nih.gov/pubmed/21681857
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/humu.21553
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