Glucocerebrosidase gene therapy prevents α-synucleinopathy of midbrain dopamine neurons

Glucocerebrosidase gene therapy prevents α-synucleinopathy of midbrain dopamine neurons

Diminished lysosomal function can lead to abnormal cellular accumulation of specific proteins, including α-synuclein, contributing to disease pathogenesis of vulnerable neurons in Parkinson's disease (PD) and related α-synucleinopathies. GBA1 encodes for the lysosomal hydrolase glucocerebrosida...

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Bibliographic Details
Main Authors: Emily M. Rocha, Gaynor A. Smith, Eric Park, Hongmei Cao, Eilish Brown, Melissa A. Hayes, Jonathan Beagan, Jesse R. McLean, Sarah C. Izen, Eduardo Perez-Torres, Penelope J. Hallett, Ole Isacson
Format: Article
Language:English
Published: Elsevier 2015-10-01
Series:Neurobiology of Disease
Subjects:
GBA
α-Synuclein
Parkinson's disease
Glucocerebrosidase
Autophagy
Gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996115300504

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http://www.sciencedirect.com/science/article/pii/S0969996115300504

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