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Improvement of diagnostic yield in carbamoylphosphate synthetase 1 (CPS1) molecular genetic investigation by RNA sequencing

Carbamoylphosphate synthetase 1 (CPS1) deficiency is a rare inborn error of metabolism leading often to neonatal onset hyperammonemia with coma and high mortality. The biochemical features of the disease are nonspecific and cannot distinguish this condition from other defects of the urea cycle, name...

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Detalhes bibliográficos
Publicado no:JIMD Rep
Main Authors: Isler, Jasmine, Rüfenacht, Véronique, Gemperle, Corinne, Allegri, Gabriella, Häberle, Johannes
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley & Sons, Inc. 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7052687/
https://ncbi.nlm.nih.gov/pubmed/32154057
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jmd2.12091
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