Therapeutics in the Pipeline Targeting α-Synuclein for Parkinson's Disease

The localization of alpha-synuclein in the endocytic pathway

(2021) Mohammad A.A. Fakhree et al.   NEUROSCIENCE

A randomized single and multiple ascending dose study in healthy volunteers of LTI‐291, a centrally penetrant glucocerebrosidase activator

(2021) Jonas M. Heijer et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Novel targeted therapies for Parkinson’s disease

(2021) Theodora Ntetsika et al.   MOLECULAR MEDICINE

Blood-brain barrier opening with focused ultrasound in Parkinson’s disease dementia

(2021) Carmen Gasca-Salas et al.   Nature Communications

α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s disease

(2021) Tracy A. Cole et al.   JCI Insight

Perampanel Inhibits α‐Synuclein Transmission in Parkinson's Disease Models

(2021) Jun Ueda et al.   MOVEMENT DISORDERS

LRRK2 at the Crossroad of Aging and Parkinson’s Disease

(2021) Eun-Mi Hur et al.   Genes

Antioxidant Therapeutics in Parkinson’s Disease: Current Challenges and Opportunities

(2021) Ana Patricia Duarte-Jurado et al.   Antioxidants

Conservative iron chelation for neurodegenerative diseases such as Parkinson’s disease and amyotrophic lateral sclerosis

(2020) David Devos et al.   JOURNAL OF NEURAL TRANSMISSION

The process of Lewy body formation, rather than simply α-synuclein fibrillization, is one of the major drivers of neurodegeneration

(2020) Anne-Laure Mahul-Mellier et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Translation of the intrinsically disordered protein α-synuclein is inhibited by a small molecule targeting its structured mRNA

(2020) Peiyuan Zhang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Inhibition of synucleinopathic seeding by rationally designed inhibitors

(2020) Smriti Sangwan et al.   eLife

Ambroxol for the Treatment of Patients With Parkinson Disease With and Without Glucocerebrosidase Gene Mutations

(2020) Stephen Mullin et al.   JAMA Neurology

Sphingosine-1-Phosphate Receptors Modulators Decrease Signs of Neuroinflammation and Prevent Parkinson’s Disease Symptoms in the 1-Methyl-4-Phenyl-1,2,3,6-Tetrahydropyridine Mouse Model

(2020) Élise Pépin et al.   Frontiers in Pharmacology

Protein transmission in neurodegenerative disease

(2020) Chao Peng et al.   Nature Reviews Neurology

Immunotherapies for Aging-Related Neurodegenerative Diseases—Emerging Perspectives and New Targets

(2020) Somin Kwon et al.   Neurotherapeutics

LRRK2 inhibitors induce reversible changes in nonhuman primate lungs without measurable pulmonary deficits

(2020) Marco A. S. Baptista et al.   Science Translational Medicine

Cell-to-Cell Transmission of Tau and α-Synuclein

(2020) Norihito Uemura et al.   TRENDS IN MOLECULAR MEDICINE

Exploring Sphingolipid Implications in Neurodegeneration

(2020) Alice V. Alessenko et al.   Frontiers in Neurology

Mendelian neurodegenerative disease genes involved in autophagy

(2020) Eleanna Stamatakou et al.   Cell Discovery

Progress toward the development of the small molecule equivalent of small interfering RNA

(2020) Matthew D. Disney et al.   CURRENT OPINION IN CHEMICAL BIOLOGY

Safety and immunogenicity of the α-synuclein active immunotherapeutic PD01A in patients with Parkinson's disease: a randomised, single-blinded, phase 1 trial

(2020) Dieter Volc et al.   LANCET NEUROLOGY

Transglutaminase 2 Depletion Attenuates α-Synuclein Mediated Toxicity in Mice

(2020) Jie Zhang et al.   NEUROSCIENCE

The Role of Oxidative Stress in Parkinson’s Disease

(2020) Kuo-Hsuan Chang et al.   Antioxidants

A Stearoyl–Coenzyme A Desaturase Inhibitor Prevents Multiple Parkinson Disease Phenotypes in α ‐Synuclein Mice

(2020) Silke Nuber et al.   ANNALS OF NEUROLOGY

Antisense Drugs Make Sense for Neurological Diseases

(2020) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology

Diabetes medications and risk of Parkinson’s disease: a cohort study of patients with diabetes

(2020) Ruth Brauer et al.   BRAIN

A Phase 1 Randomized Trial of Specific Active α‐Synuclein Immunotherapies PD01A and PD03A in Multiple System Atrophy

(2020) Wassilios G. Meissner et al.   MOVEMENT DISORDERS

Squalamine Restores the Function of the Enteric Nervous System in Mouse Models of Parkinson’s Disease

(2020) Christine L. West et al.   Journal of Parkinsons Disease

Anti-α-synuclein ASO delivered to monoamine neurons prevents α-synuclein accumulation in a Parkinson's disease-like mouse model and in monkeys

(2020) Diana Alarcón-Arís et al.   EBioMedicine

Targeting α-synuclein by PD03 AFFITOPE® and Anle138b rescues neurodegenerative pathology in a model of multiple system atrophy: clinical relevance

(2020) Miguel Lemos et al.   Translational Neurodegeneration

Crossing the blood-brain barrier with AAV vectors

(2020) Dan Liu et al.   METABOLIC BRAIN DISEASE

Novel antibodies detect additional α-synuclein pathology in synucleinopathies: potential development for immunotherapy

(2020) Jacqui T. Nimmo et al.   Alzheimers Research & Therapy

Efficacy of Nilotinib in Patients With Moderately Advanced Parkinson Disease

(2020) Tanya Simuni et al.   JAMA Neurology

Extended Treatment with Glial Cell Line-Derived Neurotrophic Factor in Parkinson’s Disease

(2019) Alan L. Whone et al.   Journal of Parkinsons Disease

Exosomes: biogenesis, biologic function and clinical potential

(2019) Yuan Zhang et al.   Cell and Bioscience

Transmission of α-synuclein seeds in neurodegenerative disease: recent developments

(2019) Richard J. Karpowicz et al.   LABORATORY INVESTIGATION

Beneficial effects of trehalose on striatal dopaminergic deficits in rodent and primate models of synucleinopathy in Parkinson's disease.

(2019) Patrick A. Howson et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

LRRK2 modifies α-syn pathology and spread in mouse models and human neurons

(2019) Gregor Bieri et al.   ACTA NEUROPATHOLOGICA

Parkinson's disease age at onset genome‐wide association study: Defining heritability, genetic loci, and α‐synuclein mechanisms

(2019) Cornelis Blauwendraat et al.   MOVEMENT DISORDERS

Focused ultrasound opening of the blood–brain barrier for treatment of Parkinson's disease

(2019) Peter A. LeWitt et al.   MOVEMENT DISORDERS

Trial of magnetic resonance–guided putaminal gene therapy for advanced Parkinson's disease

(2019) John D. Heiss et al.   MOVEMENT DISORDERS

FTY720 Improves Behavior, Increases Brain Derived Neurotrophic Factor and Reduces α-Synuclein Pathology in Parkinsonian GM2 +/− Mice

(2019) Guadalupe Vidal-Martinez et al.   NEUROSCIENCE

Depopulation of dense α-synuclein aggregates is associated with rescue of dopamine neuron dysfunction and death in a new Parkinson’s disease model

(2019) Michal Wegrzynowicz et al.   ACTA NEUROPATHOLOGICA

Progress of immunotherapy of anti-α-synuclein in Parkinson’s disease

(2019) Zhipeng Wang et al.   BIOMEDICINE & PHARMACOTHERAPY

α-Synuclein in Parkinson’s disease: causal or bystander?

(2019) Peter Riederer et al.   JOURNAL OF NEURAL TRANSMISSION

CSF and blood biomarkers for Parkinson's disease

(2019) Lucilla Parnetti et al.   LANCET NEUROLOGY

α-synuclein in the pathophysiology of Alzheimer’s disease

(2019) Daniel Twohig et al.   Molecular Neurodegeneration

Randomized phase I clinical trial of anti–α‐synuclein antibody BIIB054

(2019) Miroslaw Brys et al.   MOVEMENT DISORDERS

Antisense therapies for movement disorders

(2019) Daniel R. Scoles et al.   MOVEMENT DISORDERS

Blood–brain barrier opening with focused ultrasound in experimental models of Parkinson's disease

(2019) Maria Eleni Karakatsani et al.   MOVEMENT DISORDERS

Transneuronal Propagation of Pathologic α-Synuclein from the Gut to the Brain Models Parkinson’s Disease

(2019) Sangjune Kim et al.   NEURON

Amido-bridged nucleic acid (AmNA)-modified antisense oligonucleotides targeting α-synuclein as a novel therapy for Parkinson’s disease

(2019) Takuya Uehara et al.   Scientific Reports

Identification of N-linked glycans as specific mediators of neuronal uptake of acetylated α-Synuclein

(2019) Melissa Birol et al.   PLOS BIOLOGY

GBA1-associated parkinsonism

(2019) Emory Ryan et al.   CURRENT OPINION IN NEUROLOGY

Redox active metals in neurodegenerative diseases

(2019) Karla Acevedo et al.   JOURNAL OF BIOLOGICAL INORGANIC CHEMISTRY

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

(2019) Lena F. Burbulla et al.   Science Translational Medicine

Optimizing intracellular antibodies (intrabodies/nanobodies) to treat neurodegenerative disorders

(2019) Anne Messer et al.   NEUROBIOLOGY OF DISEASE

Preclinical development of a high affinity α-synuclein antibody, MEDI1341, that can enter the brain, sequester extracellular α-synuclein and attenuate α-synuclein spreading in vivo

(2019) Darren J. Schofield et al.   NEUROBIOLOGY OF DISEASE

Characterization of novel conformation-selective α-synuclein antibodies as potential immunotherapeutic agents for Parkinson's disease

(2019) Michael X. Henderson et al.   NEUROBIOLOGY OF DISEASE

Multistep Inhibition of α-Synuclein Aggregation and Toxicity in Vitro and in Vivo by Trodusquemine

(2018) Michele Perni et al.   ACS Chemical Biology

The c-Abl inhibitor, Radotinib HCl, is neuroprotective in a preclinical Parkinson’s disease mouse model

(2018) Saebom Lee et al.   HUMAN MOLECULAR GENETICS

Extracellular α-synuclein levels are regulated by neuronal activity

(2018) Kaoru Yamada et al.   Molecular Neurodegeneration

Selective α-Synuclein Knockdown in Monoamine Neurons by Intranasal Oligonucleotide Delivery: Potential Therapy for Parkinson’s Disease

(2018) Diana Alarcón-Arís et al.   MOLECULAR THERAPY

Targeted Therapies for Parkinson's Disease: From Genetics to the Clinic

(2018) S. Pablo Sardi et al.   MOVEMENT DISORDERS

Block of A1 astrocyte conversion by microglia is neuroprotective in models of Parkinson’s disease

(2018) Seung Pil Yun et al.   NATURE MEDICINE

Subcellular localization of alpha-synuclein aggregates and their interaction with membranes

(2018) Emanuela Colla et al.   Neural Regeneration Research

Familial knockin mutation of LRRK2 causes lysosomal dysfunction and accumulation of endogenous insoluble α-synuclein in neurons

(2018) Jason Schapansky et al.   NEUROBIOLOGY OF DISEASE

Reversible Conformational Conversion of α-Synuclein into Toxic Assemblies by Glucosylceramide

(2018) Friederike Zunke et al.   NEURON

MicroRNA-7 facilitates the degradation of alpha-synuclein and its aggregates by promoting autophagy

(2018) Doo Chul Choi et al.   NEUROSCIENCE LETTERS

GBA1 deficiency negatively affects physiological α-synuclein tetramers and related multimers

(2018) Sangjune Kim et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Safety and Tolerability of Multiple Ascending Doses of PRX002/RG7935, an Anti–α-Synuclein Monoclonal Antibody, in Patients With Parkinson Disease

(2018) Joseph Jankovic et al.   JAMA Neurology

β2-adrenoreceptor medications and risk of Parkinson disease

(2018) Susan Searles Nielsen et al.   ANNALS OF NEUROLOGY

USP14 inhibition corrects an in vivo model of impaired mitophagy

(2018) Joy Chakraborty et al.   EMBO Molecular Medicine

Downregulation of SNCA Expression by Targeted Editing of DNA Methylation: A Potential Strategy for Precision Therapy in PD

(2018) Boris Kantor et al.   MOLECULAR THERAPY

Noninvasive delivery of an α-synuclein gene silencing vector with magnetic resonance-guided focused ultrasound

(2018) Kristiana Xhima et al.   MOVEMENT DISORDERS

LRRK2 kinase regulates α-synuclein propagation via RAB35 phosphorylation

(2018) Eun-Jin Bae et al.   Nature Communications

The small molecule alpha-synuclein misfolding inhibitor, NPT200-11, produces multiple benefits in an animal model of Parkinson’s disease

(2018) Diana L. Price et al.   Scientific Reports

Trehalose induces autophagy via lysosomal-mediated TFEB activation in models of motoneuron degeneration

(2018) Paola Rusmini et al.   Autophagy

Endosomal Escape and Cytosolic Penetration of Macromolecules Mediated by Synthetic Delivery Agents

(2018) Dakota J. Brock et al.   BIOCONJUGATE CHEMISTRY

Lipidomic Analysis of α-Synuclein Neurotoxicity Identifies Stearoyl CoA Desaturase as a Target for Parkinson Treatment

(2018) Saranna Fanning et al.   MOLECULAR CELL

Anle138b modulates α-synuclein oligomerization and prevents motor decline and neurodegeneration in a mouse model of multiple system atrophy

(2018) Antonio Heras-Garvin et al.   MOVEMENT DISORDERS

Genotype-phenotype relations for the Parkinson's disease genes SNCA, LRRK2, VPS35: MDSGene systematic review

(2018) Joanne Trinh et al.   MOVEMENT DISORDERS

Synergistic neuroprotection by coffee components eicosanoyl-5-hydroxytryptamide and caffeine in models of Parkinson's disease and DLB

(2018) Run Yan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Poly(ADP-ribose) drives pathologic α-synuclein neurodegeneration in Parkinson’s disease

(2018) Tae-In Kam et al.   SCIENCE

Inhibiting Stearoyl-CoA Desaturase Ameliorates α-Synuclein Cytotoxicity

(2018) Benjamin M. Vincent et al.   Cell Reports

The Parkinson's progression markers initiative (PPMI) – establishing a PD biomarker cohort

(2018) Kenneth Marek et al.   Annals of Clinical and Translational Neurology

Development of an aggregate-selective, human-derived α-synuclein antibody BIIB054 that ameliorates disease phenotypes in Parkinson's disease models

(2018) Andreas Weihofen et al.   NEUROBIOLOGY OF DISEASE

Therapeutic approaches to target alpha-synuclein pathology

(2017) Patrik Brundin et al.   EXPERIMENTAL NEUROLOGY

α-synuclein aggregation and its modulation

(2017) Dhiman Ghosh et al.   INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES

Discovery of a 3-(4-Pyrimidinyl) Indazole (MLi-2), an Orally Available and Selective Leucine-Rich Repeat Kinase 2 (LRRK2) Inhibitor that Reduces Brain Kinase Activity

(2017) Jack D. Scott et al.   JOURNAL OF MEDICINAL CHEMISTRY

DJ-1 deficiency impairs autophagy and reduces alpha-synuclein phagocytosis by microglia

(2017) Yuval Nash et al.   JOURNAL OF NEUROCHEMISTRY

Glucosylsphingosine Promotes α-Synuclein Pathology in Mutant GBA-Associated Parkinson's Disease

(2017) Yumiko V. Taguchi et al.   JOURNAL OF NEUROSCIENCE

Solving the conundrum of insoluble protein aggregates

(2017) Sonia George et al.   LANCET NEUROLOGY

Neuropathological and genetic correlates of survival and dementia onset in synucleinopathies: a retrospective analysis

(2017) David J Irwin et al.   LANCET NEUROLOGY

Therapeutic potential of autophagy-enhancing agents in Parkinson’s disease

(2017) Tim E. Moors et al.   Molecular Neurodegeneration

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

Diagnosis and management of dementia with Lewy bodies

(2017) Ian G. McKeith et al.   NEUROLOGY

Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models

(2017) S. Pablo Sardi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A natural product inhibits the initiation of α-synuclein aggregation and suppresses its toxicity

(2017) Michele Perni et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

β2-Adrenoreceptor is a regulator of the α-synuclein gene driving risk of Parkinson’s disease

(2017) Shuchi Mittal et al.   SCIENCE

Oral ambroxol increases brain glucocerebrosidase activity in a nonhuman primate

(2017) Anna Migdalska-Richards et al.   SYNAPSE

Cell Biology and Pathophysiology of α-Synuclein

(2017) Jacqueline Burré et al.   Cold Spring Harbor Perspectives in Medicine

DJ-1 Inhibits α-Synuclein Aggregation by Regulating Chaperone-Mediated Autophagy

(2017) Chuan-Ying Xu et al.   Frontiers in Aging Neuroscience

c-Abl and Parkinson’s Disease: Mechanisms and Therapeutic Potential

(2017) Saurav Brahmachari et al.   Journal of Parkinsons Disease

The Synucleinopathies: Twenty Years On

(2017) Michel Goedert et al.   Journal of Parkinsons Disease

Polyethylenimine Nanoparticle-Mediated siRNA Delivery to Reduce α-Synuclein Expression in a Model of Parkinson’s Disease

(2017) Christin Helmschrodt et al.   Molecular Therapy-Nucleic Acids

LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model

(2017) Hien Tran Zhao et al.   Molecular Therapy-Nucleic Acids

The novel compound PBT434 prevents iron mediated neurodegeneration and alpha-synuclein toxicity in multiple models of Parkinson’s disease

(2017) David I. Finkelstein et al.   Acta Neuropathologica Communications

Anti-α-synuclein immunotherapy reduces α-synuclein propagation in the axon and degeneration in a combined viral vector and transgenic model of synucleinopathy

(2017) Brian Spencer et al.   Acta Neuropathologica Communications

Brain iron chelation by deferiprone in a phase 2 randomised double-blinded placebo controlled clinical trial in Parkinson’s disease

(2017) Antonio Martin-Bastida et al.   Scientific Reports

Ambroxol effects in glucocerebrosidase and α-synuclein transgenic mice

(2016) Anna Migdalska-Richards et al.   ANNALS OF NEUROLOGY

Ade novocompound targeting α-synuclein improves deficits in models of Parkinson’s disease

(2016) Wolfgang Wrasidlo et al.   BRAIN

FTY720/Fingolimod Reduces Synucleinopathy and Improves Gut Motility in A53T Mice

(2016) Guadalupe Vidal-Martínez et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

The relationship between glucocerebrosidase mutations and Parkinson disease

(2016) Anna Migdalska-Richards et al.   JOURNAL OF NEUROCHEMISTRY

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

Therapeutic Strategies and Pharmacological Tools Influencing S1P Signaling and Metabolism

(2016) Dominik Vogt et al.   MEDICINAL RESEARCH REVIEWS

First-in-human assessment of PRX002, an anti-α-synuclein monoclonal antibody, in healthy volunteers

(2016) Dale B. Schenk et al.   MOVEMENT DISORDERS

FTY720 Attenuates 6-OHDA-Associated Dopaminergic Degeneration in Cellular and Mouse Parkinsonian Models

(2016) Manru Ren et al.   NEUROCHEMICAL RESEARCH

A LRRK2-Dependent EndophilinA Phosphoswitch Is Critical for Macroautophagy at Presynaptic Terminals

(2016) Sandra-Fausia Soukup et al.   NEURON

Bifunctional Anti-Non-Amyloid Component α-Synuclein Nanobodies Are Protective In Situ

(2016) David C. Butler et al.   PLoS One

Extensive graft-derived dopaminergic innervation is maintained 24 years after transplantation in the degenerating parkinsonian brain

(2016) Wen Li et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Pathological  -synuclein transmission initiated by binding lymphocyte-activation gene 3

(2016) X. Mao et al.   SCIENCE

Mitochondrial pyruvate carrier regulates autophagy, inflammation, and neurodegeneration in experimental models of Parkinsons disease

(2016) A. Ghosh et al.   Science Translational Medicine

Nilotinib – Differentiating the Hope from the Hype

(2016) Richard K. Wyse et al.   Journal of Parkinsons Disease

Nilotinib Effects in Parkinson’s disease and Dementia with Lewy bodies

(2016) Fernando Pagan et al.   Journal of Parkinsons Disease

Implication of Alpha-Synuclein Phosphorylation at S129 in Synucleinopathies: What Have We Learned in the Last Decade?

(2016) Abid Oueslati   Journal of Parkinsons Disease

Association ofGBAMutations and the E326K Polymorphism With Motor and Cognitive Progression in Parkinson Disease

(2016) Marie Y. Davis et al.   JAMA Neurology

Anle138b Partly Ameliorates Motor Deficits Despite Failure of Neuroprotection in a Model of Advanced Multiple System Atrophy

(2016) Lisa Fellner et al.   Frontiers in Neuroscience

Is Alpha-Synuclein Loss-of-Function a Contributor to Parkinsonian Pathology? Evidence from Non-human Primates

(2016) Timothy J. Collier et al.   Frontiers in Neuroscience

Dysregulation of protein phosphatase 2A in parkinson disease and dementia with lewy bodies

(2016) Hye-Jin Park et al.   Annals of Clinical and Translational Neurology

α -synuclein conformational antibodies fused to penetratin are effective in models of Lewy body disease

(2016) Brian Spencer et al.   Annals of Clinical and Translational Neurology

Parkinson disease-linked GBA mutation effects reversed by molecular chaperones in human cell and fly models

(2016) Alvaro Sanchez-Martinez et al.   Scientific Reports

Pathological α-synuclein distribution in subjects with coincident Alzheimer’s and Lewy body pathology

(2015) Jon B. Toledo et al.   ACTA NEUROPATHOLOGICA

Chronic MPTP treatment produces hyperactivity in male mice which is not alleviated by concurrent trehalose treatment

(2015) Sherry A. Ferguson et al.   BEHAVIOURAL BRAIN RESEARCH

Ceruloplasmin activity and iron chelation treatment of patients with Parkinson’s disease

(2015) Guillaume Grolez et al.   BMC Neurology

Glucocerebrosidase activity in Parkinson’s disease with and withoutGBAmutations

(2015) Roy N. Alcalay et al.   BRAIN

Age-Related Neurodegeneration Prevention Through mTOR Inhibition: Potential Mechanisms and Remaining Questions

(2015) Jordan Jahrling et al.   CURRENT TOPICS IN MEDICINAL CHEMISTRY

Leucine-rich Repeat Kinase 2 (LRRK2) Pharmacological Inhibition Abates α-Synuclein Gene-induced Neurodegeneration

(2015) João P. L. Daher et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

shRNA targeting α-synuclein prevents neurodegeneration in a Parkinson’s disease model

(2015) Alevtina D. Zharikov et al.   JOURNAL OF CLINICAL INVESTIGATION

Parkinson's disease

(2015) Lorraine V Kalia et al.   LANCET

Targeting α-synuclein for treatment of Parkinson's disease: mechanistic and therapeutic considerations

(2015) Benjamin Dehay et al.   LANCET NEUROLOGY

Treatment with Trehalose Prevents Behavioral and Neurochemical Deficits Produced in an AAV α-Synuclein Rat Model of Parkinson’s Disease

(2015) Qing He et al.   MOLECULAR NEUROBIOLOGY

Active immunization against alpha-synuclein ameliorates the degenerative pathology and prevents demyelination in a model of multiple system atrophy

(2015) Markus Mandler et al.   Molecular Neurodegeneration

Rotenone impairs autophagic flux and lysosomal functions in Parkinson’s disease

(2015) F. Wu et al.   NEUROSCIENCE

Immunotherapeutic Approaches Targeting Amyloid-β, α-Synuclein, and Tau for the Treatment of Neurodegenerative Disorders

(2015) Elvira Valera et al.   Neurotherapeutics

Effect of selective LRRK2 kinase inhibition on nonhuman primate lung

(2015) Reina N. Fuji et al.   Science Translational Medicine

Discovery of a Pyrrolopyrimidine (JH-II-127), a Highly Potent, Selective, and Brain Penetrant LRRK2 Inhibitor

(2015) John M. Hatcher et al.   ACS Medicinal Chemistry Letters

Normalization of Overexpressed α-Synuclein Causing Parkinson's Disease By a Moderate Gene Silencing With RNA Interference

(2015) Masaki Takahashi et al.   Molecular Therapy-Nucleic Acids

The oligomer modulator anle138b inhibits disease progression in a Parkinson mouse model even with treatment started after disease onset

(2014) Johannes Levin et al.   ACTA NEUROPATHOLOGICA

Next-generation active immunization approach for synucleinopathies: implications for Parkinson’s disease clinical trials

(2014) Markus Mandler et al.   ACTA NEUROPATHOLOGICA

Targeting alpha-synuclein with a microRNA-embedded silencing vector in the rat substantia nigra: Positive and negative effects

(2014) Christina E. Khodr et al.   BRAIN RESEARCH

An Evaluation of MSDC-0160, A Prototype mTOT Modulating Insulin Sensitizer, in Patients with Mild Alzheimer’s Disease

(2014) Raj Shah et al.   Current Alzheimer Research

Transglutaminase 2 exacerbates α-synuclein toxicity in mice and yeast

(2014) Hilary Grosso et al.   FASEB JOURNAL

Inhibition of miR-34b and miR-34c enhances α-synuclein expression in Parkinson's disease

(2014) Savan Kabaria et al.   FEBS LETTERS

A Bacteriophage Capsid Protein Provides a General Amyloid Interaction Motif (GAIM) That Binds and Remodels Misfolded Protein Assemblies

(2014) Rajaraman Krishnan et al.   JOURNAL OF MOLECULAR BIOLOGY

Reducing C-Terminal-Truncated Alpha-Synuclein by Immunotherapy Attenuates Neurodegeneration and Propagation in Parkinson's Disease-Like Models

(2014) D. Games et al.   JOURNAL OF NEUROSCIENCE

Sphingosine Kinase 1 and Sphingosine-1-Phosphate in Oxidative Stress Evoked by 1-Methyl-4-Phenylpyridinium (MPP+) in Human Dopaminergic Neuronal Cells

(2014) Joanna Pyszko et al.   MOLECULAR NEUROBIOLOGY

Systemic exosomal siRNA delivery reduced alpha-synuclein aggregates in brains of transgenic mice

(2014) J. Mark Cooper et al.   MOVEMENT DISORDERS

Large-scale meta-analysis of genome-wide association data identifies six new risk loci for Parkinson's disease

(2014) Mike A Nalls et al.   NATURE GENETICS

A novel α-synuclein mutation A53E associated with atypical multiple system atrophy and Parkinson's disease-type pathology

(2014) Petra Pasanen et al.   NEUROBIOLOGY OF AGING

Immunotherapy targeting α-synuclein protofibrils reduced pathology in (Thy-1)-h[A30P] α-synuclein mice

(2014) Veronica Lindström et al.   NEUROBIOLOGY OF DISEASE

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

(2014) Franziska Richter et al.   Neurotherapeutics

Commonalities and Challenges in the Development of Clinical Trial Measures in Neurology

(2014) Jesse M. Cedarbaum et al.   Neurotherapeutics

Neuroprotective effect of the chemical chaperone, trehalose in a chronic MPTP-induced Parkinson's disease mouse model

(2014) Sumit Sarkar et al.   NEUROTOXICOLOGY

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

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

Multiple System Atrophy

(2014) Tasneem Peeraully   SEMINARS IN NEUROLOGY

iPSC-derived neurons from GBA1-associated Parkinson’s disease patients show autophagic defects and impaired calcium homeostasis

(2014) David C. Schöndorf et al.   Nature Communications

The c-Abl inhibitor, Nilotinib, protects dopaminergic neurons in a preclinical animal model of Parkinson's disease

(2014) Senthilkumar S. Karuppagounder et al.   Scientific Reports

α-Synuclein Immunotherapy Blocks Uptake and Templated Propagation of Misfolded α-Synuclein and Neurodegeneration

(2014) Hien T. Tran et al.   Cell Reports

α-Synucleinopathy associated with G51D SNCA mutation: a link between Parkinson’s disease and multiple system atrophy?

(2013) Aoife P. Kiely et al.   ACTA NEUROPATHOLOGICA

Anle138b: a novel oligomer modulator for disease-modifying therapy of neurodegenerative diseases such as prion and Parkinson’s disease

(2013) Jens Wagner et al.   ACTA NEUROPATHOLOGICA

G51D α-synuclein mutation causes a novel Parkinsonian-pyramidal syndrome

(2013) Suzanne Lesage et al.   ANNALS OF NEUROLOGY

Lewy body extracts from Parkinson disease brains trigger α-synuclein pathology and neurodegeneration in mice and monkeys

(2013) Ariadna Recasens et al.   ANNALS OF NEUROLOGY

Targeting Chelatable Iron as a Therapeutic Modality in Parkinson's Disease

(2013) David Devos et al.   ANTIOXIDANTS & REDOX SIGNALING

Prion-like spreading of pathological α-synuclein in brain

(2013) Masami Masuda-Suzukake et al.   BRAIN

Caudo-rostral brain spreading of α-synuclein through vagal connections

(2013) Ayse Ulusoy et al.   EMBO Molecular Medicine

Nilotinib reverses loss of dopamine neurons and improves motor behavior via autophagic degradation of  -synuclein in Parkinson's disease models

(2013) M. L. Hebron et al.   HUMAN MOLECULAR GENETICS

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