Substrate reduction therapy using Genz‐667161 reduces levels of pathogenic components in a mouse model of neuronopathic forms of Gaucher disease

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

Journal of neurochemistry, 2021-03, Vol.156 (5), p.692-701 [Periódico revisado por pares]

England: Blackwell Publishing Ltd

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