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Substrate Reduction Therapy Using Genz‐667161 Reduces Levels of Pathogenic Components in a Mouse Model of Neuronopathic Forms of Gaucher Disease

Shani Blumenreich,Chen Yaacobi,Ayelet Vardi,Or B. Barav,Einat B. Vitner,Hyejung Park,Bing Wang,Seng H. Cheng,Sergio P. Sardi,Anthony H. Futerman

Journal of Neurochemistry（2020）

Cited 20|Views49
Key words
Gaucher disease,glycosphingolipids,pathogenic pathways,substrate reduction therapy
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