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Gaucher Disease: Transcriptome Analyses Using Microarray or mRNA Sequencing in a Gba1 Mutant Mouse Model Treated with Velaglucerase alfa or Imiglucerase

Gaucher disease type 1, an inherited lysosomal storage disorder, is caused by mutations in GBA1 leading to defective glucocerebrosidase (GCase) function and consequent excess accumulation of glucosylceramide/glucosylsphingosine in visceral organs. Enzyme replacement therapy (ERT) with the biosimilar...

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Detalles Bibliográficos
Autores principales: Dasgupta, Nupur, Xu, You-Hai, Oh, Sunghee, Sun, Ying, Jia, Li, Keddache, Mehdi, Grabowski, Gregory A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3790783/
https://www.ncbi.nlm.nih.gov/pubmed/24124461
http://dx.doi.org/10.1371/journal.pone.0074912