Approved enzyme replacement therapies for Gaucher's disease do not help neurological symptoms. Vitner et al. used a mouse model of Gaucher's disease to show that neuronal cell death hinged on a pathway of programmed cell necrosis that involved the kinases RIPK1 (receptor-interacting protein kinase 1) and RIPK3. Mice that were deficient in RIPK3 had less neuronal loss and liver injury, improved motor coordination and a longer lifespan than mouse models with normal RIPK3 expression, suggesting that RIPK3 could be a new target for the disorder.
References
Vitner, E. B. et al. RIPK3 as a potential therapeutic target for Gaucher's disease. Nature Med. 20, 204–208 (2014)
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Harrison, C. Targeting neuronal loss in Gaucher's disease. Nat Rev Drug Discov 13, 178 (2014). https://doi.org/10.1038/nrd4270
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DOI: https://doi.org/10.1038/nrd4270