Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity

Neuronopathic glycosphingolipidoses are a sub-group of lysosomal storage disorders for which there are presently no effective therapies. Here, we evaluated the potential of substrate reduction therapy (SRT) using an inhibitor of glucosylceramide synthase (GCS) to decrease the synthesis of glucosylce...

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Vydáno v:Mol Ther
Hlavní autoři: Marshall, John, Nietupski, Jennifer B., Park, Hyejung, Cao, James, Bangari, Dinesh S., Silvescu, Cristina, Wilper, Terry, Randall, Kristen, Tietz, Drew, Wang, Bing, Ying, Xiaoyou, Leonard, John P., Cheng, Seng H.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2019
Témata:
Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6697407/
https://ncbi.nlm.nih.gov/pubmed/31208914
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.05.018
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