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A deep intronic variant is a common cause of OTC deficiency in individuals with previously negative genetic testing

Pathogenic variants in non-coding regions of genes encoding enzymes or transporters of the urea cycle can lead to urea cycle disorders (UCDs). However, not all commercially available testing platforms interrogate these regions. Here, we used a gene panel based on massively parallel sequencing (MPS)...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab Rep
Main Authors: Kumar, Runjun D., Burrage, Lindsay C., Bartos, Jan, Ali, Saima, Schmitt, Eric, Nagamani, Sandesh C.S., LeMons, Cynthia
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2021
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7809430/
https://ncbi.nlm.nih.gov/pubmed/33489762
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2020.100706
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