Differential Molecular Effects of Imiglucerase and Velaglucerase Alfa in Gaucher Disease Mice [RNA-Seq]

ABSTRACT: Gaucher disease type 1 is an inborn error of metabolic disease with the defective activity of the lysosomal enzyme acid b-glucosidase (GCase). Enzyme replacement/reconstitution therapy (ERT), infusions with purified recombinant GCases, is efficacious in reversing hematologic, hepatic, splenic, and bony disease manifestations in Gaucher type 1 patients. However, the tissue specific molecular events in Gaucher disease and their response to therapy are not known yet. To explore the molecular events underlying GCase treatment, we evaluated the tissue-specific gene expression profiles and molecular responses in our Gaucher disease mouse model, which were treated with two FDA approved commercially available GCases, imiglucerase (imig) and velaglucerase alfa (vela). Using microarray and mRNA-Seq

ORGANISM(S): Mus musculus

SUBMITTER: nupur dasgupta 

PROVIDER: E-GEOD-44640 | biostudies-arrayexpress |

REPOSITORIES: biostudies-arrayexpress