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 glucocerebrosidase (G...

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Hlavní autoři:	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
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Elsevier 2015-10-01
Edice:	Neurobiology of Disease
Témata:	GBA α-Synuclein Parkinson's disease Glucocerebrosidase Autophagy Gene therapy
On-line přístup:	http://www.sciencedirect.com/science/article/pii/S0969996115300504
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