A new glucocerebrosidase chaperone reduces α-synuclein and glycolipid levels in iPSC-derived dopaminergic neurons from patients with Gaucher disease and parkinsonism

Journal of Neuroscience

Volumn 36, Issue 28, 2016, Pages 7441-7452

  Aflaki, Elma ^a   Borger, Daniel K ^a   Moaven, Nima ^a   Stubblefield, Barbara K ^a   Rogers, Steven A ^b   Patnaik, Samarjit ^c   Schoenen, Frank J ^b   Westbroek, Wendy ^a   Zheng, Wei ^c   Sullivan, Patricia ^d   Fujiwara, Hideji ^e   Sidhu, Rohini ^e   Khaliq, Zayd M ^f   Lopez, Grisel J ^a   Goldstein, David S ^d   Ory, Daniel S ^e   Marugan, Juan ^c   Sidransky, Ellen ^a  

^a NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

^b UNIVERSITY OF KANSAS   (United States)

^c NATIONAL INSTITUTES OF HEALTH   (United States)

^d Clinical Neurocardiology Section   (United States)

^e WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^f NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

Author keywords

Dopaminergic neurons; Glucocerebrosidase; Induced pluripotent stem cells; Parkinsonism; Pharmacological chaperone; synuclein

Indexed keywords

ALPHA SYNUCLEIN; CHAPERONE; DOPAMINE TRANSPORTER; GLUCOSYLCERAMIDASE; GLYCOLIPID; ACETANILIDE DERIVATIVE; BENZAMIDE DERIVATIVE; BETA GLUCOSIDASE; CATECHOLAMINE; GBA2 PROTEIN, HUMAN; GLUCOSYLCERAMIDE; LYSOSOME ASSOCIATED MEMBRANE PROTEIN 2; NCGC607;

ARTICLE; CELL DIFFERENTIATION; CONTROLLED STUDY; ELECTROPHYSIOLOGICAL PROCEDURES; ELECTROSPRAY; ENZYME ACTIVITY; GAUCHER DISEASE; GENE MUTATION; HIGH THROUGHPUT SCREENING; HUMAN; HUMAN CELL; IMMUNOBLOTTING; IMMUNOFLUORESCENCE; MASS SPECTROMETRY; PARKINSONISM; PATHOGENESIS; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; REAL TIME POLYMERASE CHAIN REACTION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; RISK FACTOR; RNA ISOLATION; WESTERN BLOTTING; ANTAGONISTS AND INHIBITORS; DOPAMINERGIC NERVE CELL; DRUG EFFECTS; FEMALE; GENETICS; INDUCED PLURIPOTENT STEM CELL; INHIBITORY POSTSYNAPTIC POTENTIAL; MACROPHAGE; MALE; MEMBRANE POTENTIAL; METABOLISM; MUTATION; PATCH CLAMP TECHNIQUE; PATHOLOGY;

ACETANILIDES; ALPHA-SYNUCLEIN; BENZAMIDES; BETA-GLUCOSIDASE; CATECHOLAMINES; CELL DIFFERENTIATION; DOPAMINERGIC NEURONS; FEMALE; GAUCHER DISEASE; GLUCOSYLCERAMIDES; GLYCOLIPIDS; HUMANS; INDUCED PLURIPOTENT STEM CELLS; INHIBITORY POSTSYNAPTIC POTENTIALS; LYSOSOMAL-ASSOCIATED MEMBRANE PROTEIN 2; MACROPHAGES; MALE; MEMBRANE POTENTIALS; MUTATION; PARKINSONIAN DISORDERS; PATCH-CLAMP TECHNIQUES;

EID: 84978826732     PISSN: 02706474     EISSN: 15292401     Source Type: Journal    
DOI: 10.1523/JNEUROSCI.0636-16.2016     Document Type: Article

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