Conditional expression of human acid β-glucosidase improves the visceral phenotype in a Gaucher disease mouse model

The reversibility and regression of histological and biochemical findings in a mouse model of Gaucher disease (4L/PS-NA) was evaluated using a liver-enriched activator protein promoter control of a tetracycline-controlled transcriptional activation-responsive human acid β-glucosidase (hGCase) transg...

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Bibliographic Details
Main Authors: Ying Sun, Brian Quinn, You-Hai Xu, Tatyana Leonova, David P. Witte, Gregory A. Grabowski
Format: Article
Language:English
Published: Elsevier 2006-10-01
Series:Journal of Lipid Research
Online Access:http://www.sciencedirect.com/science/article/pii/S0022227520434030