Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease

Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease

The neuropathic glycosphingolipidoses are a subgroup of lysosomal storage disorders for which there are no effective therapies. A potential approach is substrate reduction therapy using inhibitors of glucosylceramide synthase (GCS) to decrease the synthesis of glucosylceramide and related glycosphin...

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Bibliographic Details
Main Authors: Ashe, Karen M., Bangari, Dinesh, Li, Lingyun, Cabrera-Salazar, Mario A., Bercury, Scott D., Nietupski, Jennifer B., Cooper, Christopher G. F., Aerts, Johannes M. F. G., Lee, Edward R., Copeland, Diane P., Cheng, Seng H., Scheule, Ronald K., Marshall, John
Format: Online
Language:English
Published: Public Library of Science 2011
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3126858/

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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3126858/

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