Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease.

Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease.

Neuropathic Gaucher disease (nGD), also known as type 2 or type 3 Gaucher disease, is caused by a deficiency of the enzyme glucocerebrosidase (GC). This deficiency impairs the degradation of glucosylceramide (GluCer) and glucosylsphingosine (GluSph), leading to their accumulation in the brains of pa...

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Bibliographic Details
Main Authors: Mario A Cabrera-Salazar, Matthew Deriso, Scott D Bercury, Lingyun Li, John T Lydon, William Weber, Nilesh Pande, Mandy A Cromwell, Diane Copeland, John Leonard, Seng H Cheng, Ronald K Scheule
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2012-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3422338?pdf=render

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http://europepmc.org/articles/PMC3422338?pdf=render

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