A New Glucocerebrosidase Chaperone Reduces &#945;-Synuclein and Glycolipid Levels in iPSC-Derived Dopaminergic Neurons from Patients with Gaucher Disease and Parkinsonism.

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

A New Glucocerebrosidase Chaperone Reduces α-Synuclein and Glycolipid Levels in iPSC-Derived Dopaminergic Neurons from Patients with Gaucher Disease and Parkinsonism.

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

Afiliación

Aflaki E; Section of Molecular Neurogenetics, National Human Genome Research Institute.
Borger DK; Section of Molecular Neurogenetics, National Human Genome Research Institute.
Moaven N; Section of Molecular Neurogenetics, National Human Genome Research Institute.
Stubblefield BK; Section of Molecular Neurogenetics, National Human Genome Research Institute.
Rogers SA; University of Kansas Specialized Chemistry Center, University of Kansas, Lawrence, Kansas 66047, and.
Patnaik S; National Center for Advancing Translational Sciences.
Schoenen FJ; University of Kansas Specialized Chemistry Center, University of Kansas, Lawrence, Kansas 66047, and.
Westbroek W; Section of Molecular Neurogenetics, National Human Genome Research Institute.
Zheng W; National Center for Advancing Translational Sciences.
Sullivan P; Clinical Neurocardiology Section, and.
Fujiwara H; Diabetic Cardiovascular Disease Center and Department of Medicine, Washington University School of Medicine, St. Louis, Missouri 63110.
Sidhu R; Diabetic Cardiovascular Disease Center and Department of Medicine, Washington University School of Medicine, St. Louis, Missouri 63110.
Khaliq ZM; Cellular Neurophysiology Unit, National Institute of Neurological Disease and Stroke, National Institutes of Health, Bethesda, Maryland 20892.
Lopez GJ; Section of Molecular Neurogenetics, National Human Genome Research Institute.
Goldstein DS; Clinical Neurocardiology Section, and.
Ory DS; Diabetic Cardiovascular Disease Center and Department of Medicine, Washington University School of Medicine, St. Louis, Missouri 63110.
Marugan J; National Center for Advancing Translational Sciences.
Sidransky E; Section of Molecular Neurogenetics, National Human Genome Research Institute, sidranse@mail.nih.gov.

J Neurosci ; 36(28): 7441-52, 2016 07 13.

Article en En | MEDLINE | ID: mdl-27413154

Asunto(s)

Neuronas Dopaminérgicas/metabolismo; Enfermedad de Gaucher/patología; Glucosilceramidas/antagonistas & inhibidores; Glucolípidos/metabolismo; Células Madre Pluripotentes Inducidas/efectos de los fármacos; Trastornos Parkinsonianos/patología; alfa-Sinucleína/metabolismo; Acetanilidas/farmacología; Benzamidas/farmacología; Catecolaminas/metabolismo; Diferenciación Celular/genética; Neuronas Dopaminérgicas/efectos de los fármacos; Femenino; Glucosilceramidasa; Glucosilceramidas/metabolismo; Humanos; Células Madre Pluripotentes Inducidas/metabolismo; Potenciales Postsinápticos Inhibidores/efectos de los fármacos; Potenciales Postsinápticos Inhibidores/genética; Proteína 2 de la Membrana Asociada a los Lisosomas/metabolismo; Macrófagos/efectos de los fármacos; Macrófagos/metabolismo; Masculino; Potenciales de la Membrana/efectos de los fármacos; Potenciales de la Membrana/genética; Mutación/genética; Técnicas de Placa-Clamp; beta-Glucosidasa/genética

Palabras clave

dopaminergic neurons; glucocerebrosidase; induced pluripotent stem cells; parkinsonism; pharmacological chaperone; α-synuclein

Texto completo

Imprimir

XML

PubMed Links

Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Glucolípidos / Trastornos Parkinsonianos / Alfa-Sinucleína / Células Madre Pluripotentes Inducidas / Neuronas Dopaminérgicas / Enfermedad de Gaucher / Glucosilceramidas Tipo de estudio: Prognostic_studies / Risk_factors_studies Límite: Female / Humans / Male Idioma: En Revista: J Neurosci Año: 2016 Tipo del documento: Article

Texto completo

Imprimir

XML

PubMed Links

Buscar en Google