A genetic model of substrate deprivation therapy for a glycosphingolipid storage disorder

Inherited defects in the degradation of glycosphingolipids (GSLs) cause a group of severe diseases known as GSL storage disorders. There are currently no effective treatments for the majority of these disorders. We have explored a new treatment paradigm, substrate deprivation therapy, by constructin...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Liu, Yujing, Wada, Ryuichi, Kawai, Hiromichi, Sango, Kazunori, Deng, Chuxia, Tai, Tadashi, McDonald, Michael P., Araujo, Kristlyn, Crawley, Jacqueline N., Bierfreund, Uwe, Sandhoff, Konrad, Suzuki, Kinuko, Proia, Richard L.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society for Clinical Investigation 1999
Pynciau:
Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC408106/
https://ncbi.nlm.nih.gov/pubmed/10021458
Tagiau: Ychwanegu Tag
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