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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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Main Authors: | , , , , , , , , , , , |
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格式: | Artigo |
語言: | Inglês |
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Public Library of Science
2012
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主題: | |
在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3422338/ https://ncbi.nlm.nih.gov/pubmed/22912851 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0043310 |
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