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Emerging therapeutic targets for Gaucher disease

Einat B Vitner1, Ayelet Vardi, Timothy M Cox

  • 1Weizmann Institute of Science, Department of Biological Chemistry , Rehovot 76100 , Israel +972 8 9342353 ; +972 8 9344112 ; einat.vitner@weizmann.ac.il.

Expert Opinion on Therapeutic Targets
|November 23, 2014
PubMed
Summary

New treatments targeting neuroinflammation pathways, like receptor-interacting protein kinase-3 (RIP3), may help neuronopathic Gaucher disease (nGD) patients. This approach offers hope beyond current therapies that don't address neurological symptoms.

Keywords:
Gaucher diseaseRIP3cell deathglucocerebrosidase 1glucocerebrosidase 2necroptosisneuroinflammationpathogenesis

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