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Type 2 Gaucher Disease in an Infant Despite a Normal Maternal Glucocerebrosidase gene

Gaucher disease (GD) is a recessively inherited autosomal lysosomal storage disease, the most severe of which is type 2, an acute neuronopathic form. We report an affected infant who inherited one mutant allele, Arg257Gln (c.887G>A; p.Arg296Gln) from his father, while the second, Gly202Arg (c.721...

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Dettagli Bibliografici
Pubblicato in:Am J Med Genet A
Autori principali: Hagege, Ermias, Grey, Richard J, Lopez, Grisel, Lal, Tamanna Roshan, Sidransky, Ellen, Tayebi, Nahid
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2017
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5787391/
https://ncbi.nlm.nih.gov/pubmed/29091352
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajmg.a.38487
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