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Severe phenotype of ATP6AP1‐CDG in two siblings with a novel mutation leading to a differential tissue‐specific ATP6AP1 protein pattern, cellular oxidative stress and hepatic copper accumulation

Congenital disorders of glycosylation (CDG) represent a wide range of >140 inherited metabolic diseases, continually expanding not only with regards to the number of newly identified causative genes, but also the heterogeneity of the clinical and molecular presentations within each subtype. The d...

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Dettagli Bibliografici
Pubblicato in:J Inherit Metab Dis
Autori principali: Ondruskova, Nina, Honzik, Tomas, Vondrackova, Alzbeta, Stranecky, Viktor, Tesarova, Marketa, Zeman, Jiri, Hansikova, Hana
Natura: Artigo
Lingua:Inglês
Pubblicazione: John Wiley & Sons, Inc. 2020
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7383996/
https://ncbi.nlm.nih.gov/pubmed/32216104
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jimd.12237
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