Glial Cell Line-Derived Neurotrophic Factor Gene Delivery in Parkinson's Disease: A Delicate Balance between Neuroprotection, Trophic Effects, and Unwanted Compensatory Mechanisms

Toxic effects of human and rodent variants of alpha-synucleinin vivo

(2017) Natalie Landeck et al.   EUROPEAN JOURNAL OF NEUROSCIENCE

Clinical tests of neurotrophic factors for human neurodegenerative diseases, part 2: Where do we stand and where must we go next?

(2017) Raymond T. Bartus et al.   NEUROBIOLOGY OF DISEASE

Clinical tests of neurotrophic factors for human neurodegenerative diseases, part 1: Where have we been and what have we learned?

(2017) Raymond T. Bartus et al.   NEUROBIOLOGY OF DISEASE

How can rAAV-α-synuclein and the fibril α-synuclein models advance our understanding of Parkinson's disease?

(2016) Laura A. Volpicelli-Daley et al.   JOURNAL OF NEUROCHEMISTRY

Developing therapeutically more efficient Neurturin variants for treatment of Parkinson's disease

(2016) Pia Runeberg-Roos et al.   NEUROBIOLOGY OF DISEASE

Long-term controlled GDNF over-expression reduces dopamine transporter activity without affecting tyrosine hydroxylase expression in the rat mesostriatal system

(2016) Pedro Barroso-Chinea et al.   NEUROBIOLOGY OF DISEASE

Is Axonal Degeneration a Key Early Event in Parkinson’s Disease?

(2016) Zuzanna Kurowska et al.   Journal of Parkinsons Disease

A regulatable AAV vector mediating GDNF biological effects at clinically-approved sub-antimicrobial doxycycline doses

(2016) Abdelwahed Chtarto et al.   Molecular Therapy-Methods & Clinical Development

Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: A double-blind, randomized, controlled trial

(2015) C. Warren Olanow et al.   ANNALS OF NEUROLOGY

GDNF-Ret signaling in midbrain dopaminergic neurons and its implication for Parkinson disease

(2015) Edgar R. Kramer et al.   FEBS LETTERS

α-Synuclein strains cause distinct synucleinopathies after local and systemic administration

(2015) W. Peelaerts et al.   NATURE

Identification of a Vav2-dependent mechanism for GDNF/Ret control of mesolimbic DAT trafficking

(2015) Shuyong Zhu et al.   NATURE NEUROSCIENCE

GDNF is not required for catecholaminergic neuron survival in vivo

(2015) Jaakko Kopra et al.   NATURE NEUROSCIENCE

Longitudinal follow-up and characterization of a robust rat model for Parkinson's disease based on overexpression of alpha-synuclein with adeno-associated viral vectors

(2015) Anke Van der Perren et al.   NEUROBIOLOGY OF AGING

Post-mortem assessment of the short and long-term effects of the trophic factor neurturin in patients with α-synucleinopathies

(2015) R.T. Bartus et al.   NEUROBIOLOGY OF DISEASE

GDNF-based therapies, GDNF-producing interneurons, and trophic support of the dopaminergic nigrostriatal pathway. Implications for Parkinson’s disease

(2015) Xavier d’Anglemont de Tassigny et al.   Frontiers in Neuroanatomy

GDNF Overexpression from the Native Locus Reveals its Role in the Nigrostriatal Dopaminergic System Function

(2015) Anmol Kumar et al.   PLoS Genetics

Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

(2014) Marc Tardieu et al.   HUMAN GENE THERAPY

Formation of α-synuclein Lewy neurite–like aggregates in axons impedes the transport of distinct endosomes

(2014) Laura A. Volpicelli-Daley et al.   MOLECULAR BIOLOGY OF THE CELL

Pharmacologically controlled, discontinuous GDNF gene therapy restores motor function in a rat model of Parkinson's disease

(2014) Julia Tereshchenko et al.   NEUROBIOLOGY OF DISEASE

Disease duration and the integrity of the nigrostriatal system in Parkinson’s disease

(2013) Jeffrey H. Kordower et al.   BRAIN

Regeneration of the MPTP-Lesioned Dopaminergic System after Convection-Enhanced Delivery of AAV2-GDNF

(2013) A. P. Kells et al.   JOURNAL OF NEUROSCIENCE

Parkinson's Disease Gene Therapy: Success by Design Meets Failure by Efficacy

(2013) Raymond T Bartus et al.   MOLECULAR THERAPY

Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients

(2013) R. T. Bartus et al.   NEUROLOGY

Axon degeneration in Parkinson's disease

(2012) Robert E. Burke et al.   EXPERIMENTAL NEUROLOGY

GDNF Is Predominantly Expressed in the PV+ Neostriatal Interneuronal Ensemble in Normal Mouse and after Injury of the Nigrostriatal Pathway

(2012) M. Hidalgo-Figueroa et al.   JOURNAL OF NEUROSCIENCE

Impaired neurotransmission caused by overexpression of  -synuclein in nigral dopamine neurons

(2012) M. Lundblad et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Long-Term Follow-Up After Gene Therapy for Canavan Disease

(2012) P. Leone et al.   Science Translational Medicine

 -Synuclein-Induced Down-Regulation of Nurr1 Disrupts GDNF Signaling in Nigral Dopamine Neurons

(2012) M. Decressac et al.   Science Translational Medicine

GDNF fails to exert neuroprotection in a rat  -synuclein model of Parkinson's disease

(2011) M. Decressac et al.   BRAIN

Heparan sulfate proteoglycan syndecan-3 is a novel receptor for GDNF, neurturin, and artemin

(2011) Maxim M. Bespalov et al.   JOURNAL OF CELL BIOLOGY

AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

(2011) Peter A LeWitt et al.   LANCET NEUROLOGY

Efficient Gene Therapy for Parkinson's Disease Using Astrocytes as Hosts for Localized Neurotrophic Factor Delivery

(2011) Anja Drinkut et al.   MOLECULAR THERAPY

Is GDNF beneficial in Parkinson disease?

(2011) Barry J. Hoffer et al.   Nature Reviews Neurology

SNARE protein redistribution and synaptic failure in a transgenic mouse model of Parkinson’s disease

(2010) Pablo Garcia-Reitböck et al.   BRAIN

The Differential Axonal Degradation of Ret Accounts for Cell-Type-Specific Function of Glial Cell Line-Derived Neurotrophic Factor as a Retrograde Survival Factor

(2010) C. C. Tsui et al.   JOURNAL OF NEUROSCIENCE

Characterization of the Intracellular Localization, Processing, and Secretion of Two Glial Cell Line-Derived Neurotrophic Factor Splice Isoforms

(2010) L. Lonka-Nevalaita et al.   JOURNAL OF NEUROSCIENCE

Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial

(2010) William J Marks et al.   LANCET NEUROLOGY

α-Synuclein Expression in Rat Substantia Nigra Suppresses Phospholipase D2 Toxicity and Nigral Neurodegeneration

(2010) Oleg S Gorbatyuk et al.   MOLECULAR THERAPY

Bioactivity of AAV2-neurturin gene therapy (CERE-120): Differences between Parkinson's disease and nonhuman primate brains

(2010) Raymond T. Bartus et al.   MOVEMENT DISORDERS

Enhanced dopamine transporter activity in middle-aged Gdnf heterozygous mice

(2010) Ofelia M. Littrell et al.   NEUROBIOLOGY OF AGING

Increased Expression of α-Synuclein Reduces Neurotransmitter Release by Inhibiting Synaptic Vesicle Reclustering after Endocytosis

(2010) Venu M. Nemani et al.   NEURON

 -Synuclein Promotes SNARE-Complex Assembly in Vivo and in Vitro

(2010) J. Burre et al.   SCIENCE

Brain α-synuclein accumulation in multiple system atrophy, Parkinson's disease and progressive supranuclear palsy: a comparative investigation

(2009) Junchao Tong et al.   BRAIN

Functional Effects of AAV2-GDNF on the Dopaminergic Nigrostriatal Pathway in Parkinsonian Rhesus Monkeys

(2009) Jamie L. Eberling et al.   HUMAN GENE THERAPY

Clinically Relevant Effects of Convection-Enhanced Delivery of AAV2-GDNF on the Dopaminergic Nigrostriatal Pathway in Aged Rhesus Monkeys

(2009) Louisa C. Johnston et al.   HUMAN GENE THERAPY

Safety Evaluation of AAV2-GDNF Gene Transfer into the Dopaminergic Nigrostriatal Pathway in Aged and Parkinsonian Rhesus Monkeys

(2009) Xiaomin Su et al.   HUMAN GENE THERAPY

Reversible neurochemical changes mediated by delayed intrastriatal glial cell line-derived neurotrophic factor gene delivery in a partial Parkinson's disease rat model

(2009) Xin Yang et al.   JOURNAL OF GENE MEDICINE

Nigrostriatal rAAV-mediated GDNF Overexpression Induces Robust Weight Loss in a Rat Model of Age-related Obesity

(2009) Fredric P Manfredsson et al.   MOLECULAR THERAPY

Mutant LRRK2 R1441G BAC transgenic mice recapitulate cardinal features of Parkinson's disease

(2009) Yanping Li et al.   NATURE NEUROSCIENCE

EXPRESSION, BIOACTIVITY, AND SAFETY 1 YEAR AFTER ADENO-ASSOCIATED VIRAL VECTOR TYPE 2–MEDIATED DELIVERY OF NEURTURIN TO THE MONKEY NIGROSTRIATAL SYSTEM SUPPORT CERE-120 FOR PARKINSON'S DISEASE

(2009) Christopher D. Herzog et al.   NEUROSURGERY

Differential Transgene Expression Profiles in Rat Brain, Using rAAV2/1 Vectors with Tetracycline-Inducible and Cytomegalovirus Promoters

(2008) Olivier Bockstael et al.   HUMAN GENE THERAPY

Tyrosine hydroxylase activity is regulated by two distinct dopamine-binding sites

(2008) Sarah L. Gordon et al.   JOURNAL OF NEUROCHEMISTRY

Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2–neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial

(2008) William J Marks et al.   LANCET NEUROLOGY

Transgene Expression, Bioactivity, and Safety of CERE-120 (AAV2-Neurturin) Following Delivery to the Monkey Striatum

(2008) Christopher D Herzog et al.   MOLECULAR THERAPY

Expression of Lewy body protein septin 4 in postmortem brain of Parkinson's disease and control subjects

(2008) Lina Shehadeh et al.   MOVEMENT DISORDERS

GDNF and GFRα: a versatile molecular complex for developing neurons

(2008) Gustavo Paratcha et al.   TRENDS IN NEUROSCIENCES

Expression of GDNF transgene in astrocytes improves cognitive deficits in aged rats

(2007) M. Pertusa et al.   NEUROBIOLOGY OF AGING