Article
Activation of β-Glucocerebrosidase Reduces Pathological α-Synuclein and Restores Lysosomal Function in Parkinson's Patient Midbrain Neurons.
The Journal of neuroscience : the official journal of the Society for Neuroscience - 20 Jul 2016
Mazzulli Joseph R, Zunke Friederike, Tsunemi Taiji, Toker Nicholas J, Jeon Sohee, Burbulla Lena F, Patnaik Samarjit, Sidransky Ellen, Marugan Juan J, Sue Carolyn M, Krainc Dimitri
Abstract excerpt
UNLABELLED: Parkinson's disease (PD) is characterized by the accumulation of α-synuclein (α-syn) within Lewy body inclusions in the nervous system. There are currently no disease-modifying therapies capable of reducing α-syn inclusions in PD. Recent data has indicated that loss-of-function mutations in the GBA1 gene that encodes lysosomal β-glucocerebrosidase (GCase) represent an important risk factor for PD, and...
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