A GCase Chaperone Improves Motor Function in a Mouse Model of Synucleinopathy

Mutation of the lysosomal hydrolase acid-β-glucosidase (GCase), which leads to reduced GCase activity, is one of the most frequent genetic risk factors for Parkinson’s disease (PD) and promotes α-synuclein accumulation in the brain, a hallmark of PD and other synucleinopathies. Whether targeting GCa...

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Veröffentlicht in:	Neurotherapeutics 2014-10, Vol.11 (4), p.840-856
Hauptverfasser:	Richter, Franziska, Fleming, Sheila M., Watson, Melanie, Lemesre, Vincent, Pellegrino, Lee, Ranes, Brian, Zhu, Chunni, Mortazavi, Farzad, Mulligan, Caitlin K., Sioshansi, Pedrom C., Hean, Sindalana, De La Rosa, Krystal, Khanna, Richie, Flanagan, John, Lockhart, David J., Wustman, Brandon A., Clark, Sean W., Chesselet, Marie-Françoise
Format:	Artikel
Sprache:	eng
Schlagworte:	alpha-Synuclein - metabolism Animals beta-Glucosidase - antagonists & inhibitors Biomedical and Life Sciences Biomedicine Brain - drug effects Brain - enzymology Brain - metabolism Disease Models, Animal Dopaminergic Neurons - drug effects Dopaminergic Neurons - metabolism Endoplasmic reticulum Enzymes Health risk assessment Humans Imino Pyranoses - pharmacology Laboratory animals Male Mice Motor Activity - drug effects Mutation Neurobiology Neurology Neurosciences Neurosurgery Original Original Article Parkinson Disease - enzymology Parkinson Disease - prevention & control Parkinson's disease Protein Aggregates - drug effects Protein Transport - drug effects Proteins Substantia Nigra - drug effects Substantia Nigra - metabolism Tartrates
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