Activation of β-glucocerebrosidase reduces pathological α-synuclein and restores lysosomal function in Parkinson’s patient midbrain neurons

Journal of Neuroscience

Volumn 36, Issue 29, 2016, Pages 7693-7706

  Mazzulli, Joseph R ^a,b   Zunke, Friederike ^b   Tsunemi, Taiji ^a,b   Toker, Nicholas J ^b   Jeon, Sohee ^b   Burbulla, Lena F ^a,b   Patnaik, Samarjit ^c   Sidransky, Ellen ^d   Marugan, Juan J ^c   Sue, Carolyn M ^e   Krainc, Dimitri ^a,b  

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

^b NORTHWESTERN UNIVERSITY   (United States)

^c NATIONAL INSTITUTES OF HEALTH   (United States)

^d NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

^e ROYAL NORTH SHORE HOSPITAL   (Australia)

Author keywords

Glucocerebrosidase; Induced pluripotent stem cells; Lysosomes; Parkinson s disease; Synucleinopathy; synuclein

Indexed keywords

ALPHA SYNUCLEIN; CARBOHYDRATE; GLUCOSYLCERAMIDASE; GLUCOSYLCERAMIDE; HEPATOCYTE NUCLEAR FACTOR 3BETA; HEXOSYLSPHINGOSINE; KRUPPEL LIKE FACTOR 4; LYSOSOME ENZYME; MYC PROTEIN; OCTAMER TRANSCRIPTION FACTOR 4; SPHINGOSINE; SYNAPSIN; TRANSCRIPTION FACTOR SOX2; UNCLASSIFIED DRUG; ATP13A2 PROTEIN, HUMAN; ENZYME INHIBITOR; LYSOSOME ASSOCIATED MEMBRANE PROTEIN 2; PROTON TRANSPORTING ADENOSINE TRIPHOSPHATASE; SNCA PROTEIN, HUMAN; SYNAPTOPHYSIN;

ARTICLE; CELL ASSAY; CELL DIFFERENTIATION; CELLULAR DISTRIBUTION; CONTROLLED STUDY; DOPAMINERGIC NERVE CELL; ENZYME ACTIVITY; GENE ACTIVATION; GENE INACTIVATION; GENE MUTATION; HUMAN; HUMAN CELL; IMMUNOFLUORESCENCE; IN VITRO STUDY; LEWY BODY; LIVE CELL ACTIVITY ASSAY; LYSOSOME; MESENCEPHALON; NERVE DEGENERATION; PARKINSON DISEASE; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PROTEIN DEGRADATION; PROTEIN EXPRESSION; WESTERN BLOTTING; CELL FRACTIONATION; DRUG EFFECTS; GENE EXPRESSION REGULATION; GENETICS; INDUCED PLURIPOTENT STEM CELL; METABOLISM; MUTATION; NEUROBLASTOMA; NEURODEGENERATIVE DISEASES; PATHOLOGY; TUMOR CELL LINE; ULTRASTRUCTURE;

ALPHA-SYNUCLEIN; CELL DIFFERENTIATION; CELL LINE, TUMOR; DOPAMINERGIC NEURONS; ENZYME INHIBITORS; GENE EXPRESSION REGULATION; GLUCOSYLCERAMIDASE; HUMANS; INDUCED PLURIPOTENT STEM CELLS; LYSOSOMAL-ASSOCIATED MEMBRANE PROTEIN 2; LYSOSOMES; MESENCEPHALON; MUTATION; NEUROBLASTOMA; NEURODEGENERATIVE DISEASES; PARKINSON DISEASE; PROTON-TRANSLOCATING ATPASES; SUBCELLULAR FRACTIONS; SYNAPTOPHYSIN;

EID: 84978758175     PISSN: 02706474     EISSN: 15292401     Source Type: Journal    
DOI: 10.1523/JNEUROSCI.0628-16.2016     Document Type: Article

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