Gene Therapy for Parkinson’s Disease, An Update

Effect of optogenetic modulation on entopeduncular input affects thalamic discharge and behavior in an AAV2-α-synuclein-induced hemiparkinson rat model

(2018) Hyeong Cheol Moon et al.   NEUROSCIENCE LETTERS

Long-term protective effects of AAV9-mesencephalic astrocyte-derived neurotrophic factor gene transfer in parkinsonian rats

(2017) Fei Hao et al.   EXPERIMENTAL NEUROLOGY

Exploring the potential of genome editing CRISPR-Cas9 technology

(2017) Vijai Singh et al.   GENE

Regulation of human GDNF gene expression in nigral dopaminergic neurons using a new doxycycline-regulated NTS-polyplex nanoparticle system

(2017) Armando J. Espadas-Alvarez et al.   Nanomedicine-Nanotechnology Biology and Medicine

Intracerebral injections of DNA nanoparticles encoding for a therapeutic gene provide partial neuroprotection in an animal model of neurodegeneration

(2017) David Yurek et al.   Nanomedicine-Nanotechnology Biology and Medicine

Unconventional neurotrophic factors CDNF and MANF: Structure, physiological functions and therapeutic potential

(2017) Maria Lindahl et al.   NEUROBIOLOGY OF DISEASE

Early Insights from Commercialization of Gene Therapies in Europe

(2017) Nicolas Touchot et al.   Genes

Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease

(2017) Yoshihide Sehara et al.   Human Gene Therapy Clinical Development

Titer and Product Affect the Distribution of Gene Expression after Intraputaminal Convection-Enhanced Delivery

(2017) Marina E. Emborg et al.   STEREOTACTIC AND FUNCTIONAL NEUROSURGERY

The genetic background of Parkinson's disease: current progress and future prospects

(2016) K. Kalinderi et al.   ACTA NEUROLOGICA SCANDINAVICA

Optogenetic Approaches for Controlling Seizure Activity

(2016) Jack K. Tung et al.   Brain Stimulation

Nurr1-Based Therapies for Parkinson's Disease

(2016) Jie Dong et al.   CNS Neuroscience & Therapeutics

Surgical Treatment of Parkinson’s Disease

(2016) Leo Verhagen Metman et al.   Current Treatment Options in Neurology

Combined Nurr1 and Foxa2 roles in the therapy of Parkinson's disease

(2016) S.-M. Oh et al.   EMBO Molecular Medicine

Parkinson Disease-linked Vps35 R524W Mutation Impairs the Endosomal Association of Retromer and Induces α-Synuclein Aggregation

(2016) Jordan Follett et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Tyrosine hydroxylase (TH), its cofactor tetrahydrobiopterin (BH4), other catecholamine-related enzymes, and their human genes in relation to the drug and gene therapies of Parkinson’s disease (PD): historical overview and future prospects

(2016) Toshiharu Nagatsu et al.   JOURNAL OF NEURAL TRANSMISSION

Teaching neurons to respond to placebos

(2016) Fabrizio Benedetti et al.   JOURNAL OF PHYSIOLOGY-LONDON

Chinese scientists to pioneer first human CRISPR trial

(2016) David Cyranoski   NATURE

Neuropeptide Y (NPY) as a therapeutic target for neurodegenerative diseases

(2016) Joana Duarte-Neves et al.   NEUROBIOLOGY OF DISEASE

Optogenetic stimulation of cortico-subthalamic projections is sufficient to ameliorate bradykinesia in 6-ohda lesioned mice

(2016) Teresa H. Sanders et al.   NEUROBIOLOGY OF DISEASE

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

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

DREADDs for Neuroscientists

(2016) Bryan L. Roth   NEURON

Probing pain pathways with light

(2016) Feng Wang et al.   NEUROSCIENCE

Human striatal recordings reveal abnormal discharge of projection neurons in Parkinson’s disease

(2016) Arun Singh et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Leucine-rich repeat kinase 2 and Parkinson's disease

(2016) Un-Beom Kang et al.   PROTEOMICS

Optogenetic Inhibition of the Subthalamic Nucleus Reduces Levodopa-Induced Dyskinesias in a Rat Model of Parkinson's Disease

(2016) Hyung Ho Yoon et al.   STEREOTACTIC AND FUNCTIONAL NEUROSURGERY

Spinal Plasticity and Behavior: BDNF-Induced Neuromodulation in Uninjured and Injured Spinal Cord

(2016) Sandra M. Garraway et al.   NEURAL PLASTICITY

CRISPR/Cas9: Implications for Modeling and Therapy of Neurodegenerative Diseases

(2016) Weili Yang et al.   Frontiers in Molecular Neuroscience

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

Optogenetics

(2015) Jens Duebel et al.   CURRENT OPINION IN OPHTHALMOLOGY

Global, regional, and national incidence, prevalence, and years lived with disability for 301 acute and chronic diseases and injuries in 188 countries, 1990–2013: a systematic analysis for the Global Burden of Disease Study 2013

(2015) Theo Vos et al.   LANCET

Lewy body dementias

(2015) Zuzana Walker et al.   LANCET

Pharmacogenetic stimulation of cholinergic pedunculopontine neurons reverses motor deficits in a rat model of Parkinson’s disease

(2015) Ilse S. Pienaar et al.   Molecular Neurodegeneration

Enhanced Efficacy of the CDNF/MANF Family by Combined Intranigral Overexpression in the 6-OHDA Rat Model of Parkinson's Disease

(2015) Óscar Cordero-Llana et al.   MOLECULAR THERAPY

CRISPR, the disruptor

(2015) Heidi Ledford   NATURE

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

Role of two sequence motifs of mesencephalic astrocyte-derived neurotrophic factor in its survival-promoting activity

(2015) K Mätlik et al.   Cell Death & Disease

Transgenic Expression of Human Glial Cell Line-Derived Neurotrophic Factor from Integration-Deficient Lentiviral Vectors is Neuroprotective in a Rodent Model of Parkinson's Disease

(2014) Ngoc B. Lu-Nguyen et al.   HUMAN GENE THERAPY

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial

(2014) Stéphane Palfi et al.   LANCET

The medical treatment of Parkinson disease from James Parkinson to George Cotzias

(2014) Stanley Fahn   MOVEMENT DISORDERS

DNAJC13genetic variants in parkinsonism

(2014) Emil K. Gustavsson et al.   MOVEMENT DISORDERS

Noninvasive optical inhibition with a red-shifted microbial rhodopsin

(2014) Amy S Chuong et al.   NATURE NEUROSCIENCE

Optogenetic Inactivation of the Subthalamic Nucleus Improves Forelimb Akinesia in a Rat Model of Parkinson Disease

(2014) Hyung Ho Yoon et al.   NEUROSURGERY

Cardiac optogenetics

(2013) Emilia Entcheva   AMERICAN JOURNAL OF PHYSIOLOGY-HEART AND CIRCULATORY PHYSIOLOGY

Roles of long noncoding RNAs in brain development, functional diversification and neurodegenerative diseases

(2013) Ping Wu et al.   BRAIN RESEARCH BULLETIN

An animal model mimicking pedunculopontine nucleus cholinergic degeneration in Parkinson’s disease

(2013) Ilse S. Pienaar et al.   Brain Structure & Function

AAV2-mediated striatum delivery of human CDNF prevents the deterioration of midbrain dopamine neurons in a 6-hydroxydopamine induced parkinsonian rat model

(2013) Xinmiao Ren et al.   EXPERIMENTAL NEUROLOGY

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

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

Control of Basal Ganglia Output by Direct and Indirect Pathway Projection Neurons

(2013) B. S. Freeze et al.   JOURNAL OF NEUROSCIENCE

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

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

TFEB-mediated autophagy rescues midbrain dopamine neurons from  -synuclein toxicity

(2013) M. Decressac et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

An ERcentric view of Parkinson's disease

(2013) Gabriela Mercado et al.   TRENDS IN MOLECULAR MEDICINE

Novel designer receptors to probe GPCR signaling and physiology

(2013) Jürgen Wess et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Gene therapy with AAV2-CDNF provides functional benefits in a rat model of Parkinson's disease

(2013) Susanne Bäck et al.   Brain and Behavior

Glial Cell Line-Derived Neurotrophic Factor Partially Ameliorates Motor Symptoms without Slowing Neurodegeneration in Mice with Respiratory Chain-Deficient Dopamine Neurons

(2012) Fredrik H. Sterky et al.   CELL TRANSPLANTATION

Simvastatin Induces Neuroprotection in 6-OHDA-Lesioned PC12 via the PI3K/AKT/Caspase 3 Pathway and Anti-Inflammatory Responses

(2012) Yun-Qi Xu et al.   CNS Neuroscience & Therapeutics

Comparison of the behavioural and histological characteristics of the 6-OHDA and α-synuclein rat models of Parkinson's disease

(2012) M. Decressac et al.   EXPERIMENTAL NEUROLOGY

Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease

(2012) Gabriele Mittermeyer et al.   HUMAN GENE THERAPY

Mesencephalic Astrocyte-derived Neurotrophic Factor Protects the Heart from Ischemic Damage and Is Selectively Secreted upon Sarco/endoplasmic Reticulum Calcium Depletion

(2012) Christopher C. Glembotski et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Design of a Single AAV Vector for Coexpression of TH and GCH1 to Establish Continuous DOPA Synthesis in a Rat Model of Parkinson's Disease

(2012) Erik Cederfjäll et al.   MOLECULAR THERAPY

NTS-Polyplex: a potential nanocarrier for neurotrophic therapy of Parkinson's disease

(2012) Daniel Martinez-Fong et al.   Nanomedicine-Nanotechnology Biology and Medicine

NRSF/REST neuronal deficient mice are more vulnerable to the neurotoxin MPTP

(2012) Mei Yu et al.   NEUROBIOLOGY OF AGING

Multifaces of neuropeptide Y in the brain – Neuroprotection, neurogenesis and neuroinflammation

(2012) J.O. Malva et al.   NEUROPEPTIDES

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

(2011) M. Decressac et al.   BRAIN

Signaling of Glial Cell Line-Derived Neurotrophic Factor and Its Receptor GFRα1 Induce Nurr1 and Pitx3 to Promote Survival of Grafted Midbrain-Derived Neural Stem Cells in a Rat Model of Parkinson Disease

(2011) Zhinian Lei et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

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

An optogenetic toolbox designed for primates

(2011) Ilka Diester et al.   NATURE NEUROSCIENCE

Neuroprotection by neuropeptide Y in cell and animal models of Parkinson's disease

(2011) Mickael Decressac et al.   NEUROBIOLOGY OF AGING

Properly scaled and targeted AAV2-NRTN (neurturin) to the substantia nigra is safe, effective and causes no weight loss: Support for nigral targeting in Parkinson's disease

(2011) Raymond T. Bartus et al.   NEUROBIOLOGY OF DISEASE

Viral vectors for gene delivery to the central nervous system

(2011) Thomas B. Lentz et al.   NEUROBIOLOGY OF DISEASE

The TrkB-Positive Dopaminergic Neurons are Less Sensitive to MPTP Insult in the Substantia Nigra of Adult C57/BL Mice

(2011) Yin-Xiu Ding et al.   NEUROCHEMICAL RESEARCH

TFEB Links Autophagy to Lysosomal Biogenesis

(2011) C. Settembre et al.   SCIENCE

The Yin and Yang of VEGF and PEDF: Multifaceted Neurotrophic Factors and Their Potential in the Treatment of Parkinson’s Disease

(2010) Torsten Falk et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Pathogenic Lysosomal Depletion in Parkinson's Disease

(2010) B. Dehay 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

Eight Years of Clinical Improvement in MPTP-Lesioned Primates After Gene Therapy With AAV2-hAADC

(2010) Piotr Hadaczek et al.   MOLECULAR THERAPY

A Phase I Study of Aromatic L-Amino Acid Decarboxylase Gene Therapy for Parkinson's Disease

(2010) Shin-ichi Muramatsu 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

Regulation of parkinsonian motor behaviours by optogenetic control of basal ganglia circuitry

(2010) Alexxai V. Kravitz et al.   NATURE

α-Synuclein and dopamine at the crossroads of Parkinson's disease

(2010) Lara Lourenço Venda et al.   TRENDS IN NEUROSCIENCES

Eight-year safety follow-up of coronary artery disease patients after local intracoronary VEGF gene transfer

(2009) M Hedman et al.   GENE THERAPY

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

Brain-derived neurotrophic factor in neurodegenerative diseases

(2009) Chiara Zuccato et al.   Nature Reviews Neurology

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

(2009) C. W. Christine et al.   NEUROLOGY

Optical Deconstruction of Parkinsonian Neural Circuitry

(2009) V. Gradinaru et al.   SCIENCE

Dopamine Gene Therapy for Parkinson's Disease in a Nonhuman Primate Without Associated Dyskinesia

(2009) B. Jarraya et al.   Science Translational Medicine

Armet, a UPR-upregulated protein, inhibits cell proliferation and ER stress-induced cell death

(2008) A APOSTOLOU et al.   EXPERIMENTAL CELL RESEARCH

Decreased NURR1 gene expression in patients with Parkinson's disease

(2008) Weidong Le et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

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

Phenotype, genotype, and worldwide genetic penetrance of LRRK2-associated Parkinson's disease: a case-control study

(2008) Daniel G Healy 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

Functional organization of the basal ganglia: Therapeutic implications for Parkinson's disease

(2008) Jose A. Obeso et al.   MOVEMENT DISORDERS

Engineering GPCR signaling pathways with RASSLs

(2008) Bruce R Conklin et al.   NATURE METHODS

Results from a phase I safety trial of hAADC gene therapy for Parkinson disease

(2008) J. L. Eberling et al.   NEUROLOGY

MPTP-induced deficits in striatal synaptic plasticity are prevented by glial cell line-derived neurotrophic factor expressed via an adeno-associated viral vector

(2007) Yuan-Hao Chen et al.   FASEB JOURNAL