A modulator of wild-type glucocerebrosidase improves pathogenic phenotypes in dopaminergic neuronal models of Parkinson’s disease

β-Glucocerebrosidase Modulators Promote Dimerization of β-Glucocerebrosidase and Reveal an Allosteric Binding Site

(2018) Jianbin Zheng et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

LRRK2 phosphorylation of auxilin mediates synaptic defects in dopaminergic neurons from patients with Parkinson’s disease

(2018) Maria Nguyen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Parkinson's Disease Is Not Simply a Prion Disorder

(2017) D. James Surmeier et al.   JOURNAL OF NEUROSCIENCE

AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy

(2017) Giuseppe Morabito et al.   MOLECULAR THERAPY

Dopamine oxidation mediates mitochondrial and lysosomal dysfunction in Parkinson’s disease

(2017) Lena F. Burbulla et al.   SCIENCE

Parkinson disease

(2017) Werner Poewe et al.   Nature Reviews Disease Primers

Detection of Free and Protein-Bound ortho-Quinones by Near-Infrared Fluorescence

(2016) Joseph R. Mazzulli et al.   ANALYTICAL CHEMISTRY

Design and Synthesis of Potent Quinazolines as Selective β-Glucocerebrosidase Modulators

(2016) Jianbin Zheng et al.   JOURNAL OF MEDICINAL CHEMISTRY

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

(2016) E. Aflaki et al.   JOURNAL OF NEUROSCIENCE

Activation of  -Glucocerebrosidase Reduces Pathological  -Synuclein and Restores Lysosomal Function in Parkinson's Patient Midbrain Neurons

(2016) J. R. Mazzulli et al.   JOURNAL OF NEUROSCIENCE

α-Synuclein–induced lysosomal dysfunction occurs through disruptions in protein trafficking in human midbrain synucleinopathy models

(2016) Joseph R. Mazzulli et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Selective loss of glucocerebrosidase activity in sporadic Parkinson’s disease and dementia with Lewy bodies

(2015) Davide Chiasserini et al.   Molecular Neurodegeneration

Glucocerebrosidase gene therapy prevents α-synucleinopathy of midbrain dopamine neurons

(2015) Emily M. Rocha et al.   NEUROBIOLOGY OF DISEASE

Reduced glucocerebrosidase is associated with increased α-synuclein in sporadic Parkinson’s disease

(2014) Karen E. Murphy et al.   BRAIN

Macrophage Models of Gaucher Disease for Evaluating Disease Pathogenesis and Candidate Drugs

(2014) E. Aflaki et al.   Science Translational Medicine

Glucocerebrosidase deficiency in substantia nigra of parkinson disease brains

(2012) Matthew E. Gegg et al.   ANNALS OF NEUROLOGY

Gaucher Disease Glucocerebrosidase and α-Synuclein Form a Bidirectional Pathogenic Loop in Synucleinopathies

(2011) Joseph R. Mazzulli et al.   CELL

Mitochondrial Parkin Recruitment Is Impaired in Neurons Derived from Mutant PINK1 Induced Pluripotent Stem Cells

(2011) P. Seibler et al.   JOURNAL OF NEUROSCIENCE

Dopamine neurons derived from human ES cells efficiently engraft in animal models of Parkinson’s disease

(2011) Sonja Kriks et al.   NATURE

CNS expression of glucocerebrosidase corrects  -synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy

(2011) S. P. Sardi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of β-glucosidase

(2010) Richie Khanna et al.   FEBS Journal

Mutant Parkin Impairs Mitochondrial Function and Morphology in Human Fibroblasts

(2010) Anne Grünewald et al.   PLoS One

Multicenter Analysis of Glucocerebrosidase Mutations in Parkinson's Disease

(2009) E. Sidransky et al.   NEW ENGLAND JOURNAL OF MEDICINE