High throughput screening for small molecule therapy for Gaucher disease using patient tissue as the source of mutant glucocerebrosidase.

High throughput screening for small molecule therapy for Gaucher disease using patient tissue as the source of mutant glucocerebrosidase.

Gaucher disease (GD), the most common lysosomal storage disorder, results from the inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCase). Previously, wildtype GCase was used for high throughput screening (HTS) of large collections of compounds to identify small molecule chaperones...

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Bibliographic Details
Main Authors: Ehud Goldin, Wei Zheng, Omid Motabar, Noel Southall, Jae Hyuk Choi, Juan Marugan, Christopher P Austin, Ellen Sidransky
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2012-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3260169?pdf=render

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http://europepmc.org/articles/PMC3260169?pdf=render

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