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...
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 |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2015-10-01
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Series: | Neurobiology of Disease |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S0969996115300504 |
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