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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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Dades bibliogràfiques
Publicat a:JIMD Rep
Autors principals: Isler, Jasmine, Rüfenacht, Véronique, Gemperle, Corinne, Allegri, Gabriella, Häberle, Johannes
Format: Artigo
Idioma:Inglês
Publicat: John Wiley & Sons, Inc. 2020
Matèries:
Accés en línia: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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