CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease

Gaucher disease (GD) is caused by a deficiency of glucocerebrosidase and the consequent lysosomal accumulation of unmetabolized glycolipid substrates. Enzyme-replacement therapy adequately manages the visceral manifestations of nonneuronopathic type-1 Gaucher patients, but not the brain disease in n...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Marshall, John, Sun, Ying, Bangari, Dinesh S, Budman, Eva, Park, Hyejung, Nietupski, Jennifer B, Allaire, Amy, Cromwell, Mary A, Wang, Bing, Grabowski, Gregory A, Leonard, John P, Cheng, Seng H
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4923322/
https://ncbi.nlm.nih.gov/pubmed/26948439
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.53
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