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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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
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Original Article
Acesso em linha: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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