Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses

Enhanced CNS transduction from AAV.PHP.eB infusion into the cisterna magna of older adult rats compared to AAV9

(2021) Diptaman Chatterjee et al.   GENE THERAPY

Intracerebroventricular Administration of AAV9-PHP.B SYN1-EmGFP Induces Widespread Transgene Expression in the Mouse and Monkey CNS

(2021) Adriana Galvan et al.   HUMAN GENE THERAPY

Glucocerebrosidase Gene Therapy Induces Alpha-Synuclein Clearance and Neuroprotection of Midbrain Dopaminergic Neurons in Mice and Macaques

(2021) Diego Sucunza et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

AAV Capsid-Promoter Interactions Determine CNS Cell-Selective Gene Expression In Vivo

(2020) Sara K. Powell et al.   MOLECULAR THERAPY

Aromatic L‐Amino Acid Decarboxylase Gene Therapy Enhances Levodopa Response in Parkinson's Disease

(2020) John G. Nutt et al.   MOVEMENT DISORDERS

Local cortical overexpression of human wild-type alpha-synuclein leads to increased dendritic spine density in mouse

(2020) L.M. Wagner et al.   NEUROSCIENCE LETTERS

Intra-striatal AAV2.retro administration leads to extensive retrograde transport in the rhesus macaque brain: implications for disease modeling and therapeutic development

(2020) Alison R. Weiss et al.   Scientific Reports

Noncanonical Roles of hα-syn (A53T) in the Pathogenesis of Parkinson’s Disease: Synaptic Pathology and Neuronal Aging

(2020) Qing-Jun Wang et al.   NEURAL PLASTICITY

Creation of a comprehensive training and career development approach to increase the number of neurosurgeons supported by National Institutes of Health funding

(2020) Russell R. Lonser et al.   JOURNAL OF NEUROSURGERY

Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?

(2020) Karim Bey et al.   Molecular Therapy-Methods & Clinical Development

Adeno-Associated Virus Capsid-Promoter Interactions in the Brain Translate from Rat to the Nonhuman Primate

(2020) Martin O. Bohlen et al.   HUMAN GENE THERAPY

Astrocyte-specific deletion of the transcription factor Yin Yang 1 in murine substantia nigra mitigates manganese-induced dopaminergic neurotoxicity

(2020) Edward Pajarillo et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

T cell infiltration and upregulation of MHCII in microglia leads to accelerated neuronal loss in an α-synuclein rat model of Parkinson’s disease

(2020) Meena S. Subbarayan et al.   Journal of Neuroinflammation

Protection from α-Synuclein induced dopaminergic neurodegeneration by overexpression of the mitochondrial import receptor TOM20

(2020) Briana R. De Miranda et al.   npj Parkinsons Disease

Systemic AAV vectors for widespread and targeted gene delivery in rodents

(2019) Rosemary C. Challis et al.   Nature Protocols

Brain tyrosinase overexpression implicates age-dependent neuromelanin production in Parkinson’s disease pathogenesis

(2019) Iria Carballo-Carbajal et al.   Nature Communications

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality

(2019) Eloise Hudry et al.   NEURON

Magnetic Resonance Imaging–Guided Phase 1 Trial of Putaminal AADC Gene Therapy for Parkinson's Disease

(2019) Chadwick W. Christine et al.   ANNALS OF NEUROLOGY

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

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

CLINICAL TRIAL HIGHLIGHTS: 1. GENE THERAPY FOR PARKINSON’S, 2. PHASE 3 STUDY IN FOCUS - INTEC PHARMA’S ACCORDION PILL, 3. CLINICAL TRIALS RESOURCES

(2019) Kevin McFarthing et al.   Journal of Parkinsons Disease

Targeted Transgene Activation in the Brain Tissue by Systemic Delivery of Engineered AAV1 Expressing CRISPRa

(2019) Cia-Hin Lau et al.   Molecular Therapy-Nucleic Acids

AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice

(2019) William A. Liguore et al.   MOLECULAR THERAPY

Intranasal delivery of mucoadhesive nanocarriers: a viable option for Parkinson’s disease treatment?

(2019) Saleha Rehman et al.   Expert Opinion on Drug Delivery

Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids

(2019) Qin Huang et al.   PLoS One

Adeno-Associated Virus Technologies and Methods for Targeted Neuronal Manipulation

(2019) Leila Haery et al.   Frontiers in Neuroanatomy

Ly6a Differential Expression in Blood–Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B

(2019) Ana Rita Batista et al.   HUMAN GENE THERAPY

Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS

(2019) Mariana Bravo-Hernandez et al.   NATURE MEDICINE

The C-terminal domain of LRRK2 with the G2019S mutation is sufficient to produce neurodegeneration of dopaminergic neurons in vivo

(2019) Noémie Cresto et al.   NEUROBIOLOGY OF DISEASE

A systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism

(2019) Marcus Davidsson et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency

(2018) Julie Tordo et al.   BRAIN

Widespread transduction of astrocytes and neurons in the mouse central nervous system after systemic delivery of a self-complementary AAV-PHP.B vector

(2018) Melvin Y. Rincon et al.   GENE THERAPY

More expansive gene transfer to the rat CNS: AAV PHP.EB vector dose–response and comparison to AAV PHP.B

(2018) Robert D. Dayton et al.   GENE THERAPY

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

α-Synuclein accumulation and GBA deficiency due to L444P GBA mutation contributes to MPTP-induced parkinsonism

(2018) Seung Pil Yun et al.   Molecular Neurodegeneration

Extensive transduction and enhanced spread of a modified AAV2 capsid in the non-human primate central nervous system

(2018) Jerusha Naidoo et al.   MOLECULAR THERAPY

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain

(2018) Yasunori Matsuzaki et al.   NEUROSCIENCE LETTERS

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

Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65

(2018) Eloise Hudry et al.   Molecular Therapy-Methods & Clinical Development

AAV-Mediated Progranulin Delivery to a Mouse Model of Progranulin Deficiency Causes T Cell-Mediated Toxicity

(2018) Defne A. Amado et al.   MOLECULAR THERAPY

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

(2018) Dan Wang et al.   Molecular Therapy-Methods & Clinical Development

Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates

(2018) Kousaku Ohno et al.   Molecular Therapy-Methods & Clinical Development

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

(2017) Fei Hao et al.   EXPERIMENTAL NEUROLOGY

Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders

(2017) K Bey et al.   GENE THERAPY

MR-guided parenchymal delivery of adeno-associated viral vector serotype 5 in non-human primate brain

(2017) L Samaranch et al.   GENE THERAPY

Recombinant AAV8-mediated intrastriatal gene delivery of CDNF protects rats against methamphetamine neurotoxicity

(2017) Lizheng Wang et al.   International Journal of Medical Sciences

Gene therapy approaches in the non-human primate model of Parkinson’s disease

(2017) D. Pignataro et al.   JOURNAL OF NEURAL TRANSMISSION

α-Synuclein nonhuman primate models of Parkinson’s disease

(2017) David J. Marmion et al.   JOURNAL OF NEURAL TRANSMISSION

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

Gene Therapeutic Reversal of Peripheral Olfactory Impairment in Bardet-Biedl Syndrome

(2017) Corey L. Williams et al.   MOLECULAR THERAPY

Viral vectors for therapy of neurologic diseases

(2017) Sourav R. Choudhury et al.   NEUROPHARMACOLOGY

Intracerebroventricular Delivery as a Safe, Long-Term Route of Drug Administration

(2017) Jessica L. Cohen-Pfeffer et al.   PEDIATRIC NEUROLOGY

Cellular selectivity of AAV serotypes for gene delivery in neurons and astrocytes by neonatal intracerebroventricular injection

(2017) Sean L. Hammond et al.   PLoS One

Therapeutic efficacy of AAV8-mediated intrastriatal delivery of human cerebral dopamine neurotrophic factor in 6-OHDA-induced parkinsonian rat models with different disease progression

(2017) Lizheng Wang et al.   PLoS One

Adeno-Associated Viral Vectors Serotype 8 for Cell-Specific Delivery of Therapeutic Genes in the Central Nervous System

(2017) Diego Pignataro et al.   Frontiers in Neuroanatomy

Axonal transport of AAV9 in nonhuman primate brain

(2016) F Green et al.   GENE THERAPY

Sequential Adeno-Associated Viral Vector Serotype 9–Green Fluorescent Protein Gene Transfer Causes Massive Inflammation and Intense Immune Response in Rat Striatum

(2016) Chun Yang et al.   HUMAN GENE THERAPY

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

(2016) Michaël Hocquemiller et al.   HUMAN GENE THERAPY

Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9

(2016) Joana Saraiva et al.   JOURNAL OF CONTROLLED RELEASE

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector

(2016) Sourav R Choudhury et al.   MOLECULAR THERAPY

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

Optogenetic activation of striatal cholinergic interneurons regulates L-dopa-induced dyskinesias

(2016) Tanuja Bordia et al.   NEUROBIOLOGY OF DISEASE

A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons

(2016) D. Gowanlock R. Tervo et al.   NEURON

α-Synuclein-Based Animal Models of Parkinson's Disease: Challenges and Opportunities in a New Era

(2016) Naomi P. Visanji et al.   TRENDS IN NEUROSCIENCES

Genome-Editing Technologies: Principles and Applications

(2016) Thomas Gaj et al.   Cold Spring Harbor Perspectives in Biology

Potent spinal parenchymal AAV9-mediated gene delivery by subpial injection in adult rats and pigs

(2016) Atsushi Miyanohara et al.   Molecular Therapy-Methods & Clinical Development

Towards a Non-Human Primate Model of Alpha-Synucleinopathy for Development of Therapeutics for Parkinson’s Disease: Optimization of AAV1/2 Delivery Parameters to Drive Sustained Expression of Alpha Synuclein and Dopaminergic Degeneration in Macaque

(2016) James B. Koprich et al.   PLoS One

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 signaling implemented by GM1 ganglioside; failure in Parkinson's disease and GM1-deficient murine model

(2015) Piotr Hadaczek et al.   EXPERIMENTAL NEUROLOGY

Intranasal gene delivery for treating Parkinson’s disease: overcoming the blood–brain barrier

(2015) Amirah E-E Aly et al.   Expert Opinion on Drug Delivery

Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats

(2015) J Hordeaux et al.   GENE THERAPY

Exosomes as drug delivery vehicles for Parkinson's disease therapy

(2015) Matthew J. Haney et al.   JOURNAL OF CONTROLLED RELEASE

Mutant Alpha-Synuclein Causes Age-Dependent Neuropathology in Monkey Brain

(2015) W. Yang et al.   JOURNAL OF NEUROSCIENCE

Glucocerebrosidase gene therapy prevents α-synucleinopathy of midbrain dopamine neurons

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

AAV9 supports wide-scale transduction of the CNS and TDP-43 disease modeling in adult rats

(2015) Kasey L Jackson et al.   Molecular Therapy-Methods & Clinical Development

Lack of additive role of ageing in nigrostriatal neurodegeneration triggered by α-synuclein overexpression

(2015) Mathieu Bourdenx et al.   Acta Neuropathologica Communications

Carbidopa-Based Modulation of the Functional Effect of the AAV2-hAADC Gene Therapy in 6-OHDA Lesioned Rats

(2015) Agnieszka Ciesielska et al.   PLoS One

Fractalkine Signaling Regulates the Inflammatory Response in an α-Synuclein Model of Parkinson Disease

(2015) Aaron D. Thome et al.   PLoS One

Pre-existing immunity to adeno-associated virus (AAV)2 limits transgene expression following intracerebral AAV2-based gene delivery in a 6-hydroxydopamine model of Parkinson's disease

(2014) Shorena Janelidze et al.   JOURNAL OF GENE MEDICINE

In vivo performance of a microfabricated catheter for intraparenchymal delivery

(2014) Martin L. Brady et al.   JOURNAL OF NEUROSCIENCE METHODS

Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10

(2014) Bin Yang et al.   MOLECULAR THERAPY

Alpha-synuclein-induced neurodegeneration is exacerbated in PINK1 knockout mice

(2014) Marusela Oliveras-Salvá et al.   NEUROBIOLOGY OF AGING

The influence of AAV2-mediated gene transfer of human IL-10 on neurodegeneration and immune response in a murine model of Parkinson's disease

(2014) Ilona Joniec-Maciejak et al.   Pharmacological Reports

Chronic Treatment with Novel Small Molecule Hsp90 Inhibitors Rescues Striatal Dopamine Levels but Not α-Synuclein-Induced Neuronal Cell Loss

(2014) Nikolaus R. McFarland et al.   PLoS One

Biology of adeno-associated viral vectors in the central nervous system

(2014) Giridhar Murlidharan et al.   Frontiers in Molecular Neuroscience

Mapping the AAV Capsid Host Antibody Response toward the Development of Second Generation Gene Delivery Vectors

(2014) Yu-Shan Tseng et al.   Frontiers in Immunology

Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna

(2014) Christian Hinderer et al.   Molecular Therapy-Methods & Clinical Development

Boosting chaperone-mediated autophagy in vivo mitigates α-synuclein-induced neurodegeneration

(2013) Maria Xilouri et al.   BRAIN

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

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

(2013) S J Gray et al.   GENE THERAPY

Strong Cortical and Spinal Cord Transduction After AAV7 and AAV9 Delivery into the Cerebrospinal Fluid of Nonhuman Primates

(2013) Lluis Samaranch et al.   HUMAN GENE THERAPY

Direct and Retrograde Transduction of Nigral Neurons with AAV6, 8, and 9 and Intraneuronal Persistence of Viral Particles

(2013) Karin Löw et al.   HUMAN GENE THERAPY

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

(2013) Raymond T Bartus et al.   MOLECULAR THERAPY

Enhanced neurotrophic distribution, cell signaling and neuroprotection following substantia nigral versus striatal delivery of AAV2-NRTN (CERE-120)

(2013) Christopher D. Herzog et al.   NEUROBIOLOGY OF DISEASE

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

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

Angiogenin in Parkinson Disease Models: Role of Akt Phosphorylation and Evaluation of AAV-Mediated Angiogenin Expression in MPTP Treated Mice

(2013) Trent U. Steidinger et al.   PLoS One

Pre-immunization with an Intramuscular Injection of AAV9-Human Erythropoietin Vectors Reduces the Vector-Mediated Transduction following Re-Administration in Rat Brain

(2013) Chun Yang et al.   PLoS One

Morphological and Behavioral Impact of AAV2/5-Mediated Overexpression of Human Wildtype Alpha-Synuclein in the Rat Nigrostriatal System

(2013) Sara E. Gombash et al.   PLoS One

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

Global brain delivery of neprilysin gene by intravascular administration of AAV vector in mice

(2013) Nobuhisa Iwata et al.   Scientific Reports

Functional Neuroanatomy of the Basal Ganglia

(2013) J. L. Lanciego et al.   Cold Spring Harbor Perspectives in Medicine

Subthalamic hGAD65 Gene Therapy and Striatum TH Gene Transfer in a Parkinson’s Disease Rat Model

(2013) Deyu Zheng et al.   NEURAL PLASTICITY

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

(2013) Susanne Bäck et al.   Brain and Behavior

Inactivation ofPink1Genein VivoSensitizes Dopamine-producing Neurons to 1-Methyl-4-phenyl-1,2,3,6-tetrahydropyridine (MPTP) and Can Be Rescued by Autosomal Recessive Parkinson Disease Genes,ParkinorDJ-1

(2012) M. Emdadul Haque et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Adeno-Associated Virus Type 2 Vector-Mediated Glial Cell Line-Derived Neurotrophic Factor Gene Transfer Induces Neuroprotection and Neuroregeneration in a Ubiquitin-Proteasome System Impairment Animal Model of Parkinson’s Disease

(2012) Yunlan Du et al.   Neurodegenerative Diseases

A Spirulina-Enhanced Diet Provides Neuroprotection in an α-Synuclein Model of Parkinson's Disease

(2012) Mibel M. Pabon et al.   PLoS One

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

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

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

(2011) Lluis Samaranch et al.   HUMAN GENE THERAPY

A Rat Model of Progressive Nigral Neurodegeneration Induced by the Parkinson's Disease-Associated G2019S Mutation in LRRK2

(2011) J. Dusonchet et al.   JOURNAL OF NEUROSCIENCE

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

AAV9: Over the Fence and Into the Woods …

(2011) John R Forsayeth et al.   MOLECULAR THERAPY

Interventional MRI-guided Putaminal Delivery of AAV2-GDNF for a Planned Clinical Trial in Parkinson's Disease

(2011) R Mark Richardson et al.   MOLECULAR THERAPY

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

Transfer of host-derived alpha synuclein to grafted dopaminergic neurons in rat

(2011) Jeffrey H. Kordower et al.   NEUROBIOLOGY OF DISEASE

Progressive neurodegenerative and behavioural changes induced by AAV-mediated overexpression of α-synuclein in midbrain dopamine neurons

(2011) M. Decressac et al.   NEUROBIOLOGY OF DISEASE

Progressive Neurodegeneration or Endogenous Compensation in an Animal Model of Parkinson's Disease Produced by Decreasing Doses of Alpha-Synuclein

(2011) James B. Koprich et al.   PLoS One

Gene transfer provides a practical means for safe, long-term, targeted delivery of biologically active neurotrophic factor proteins for neurodegenerative diseases

(2011) Christopher D. Herzog et al.   Drug Delivery and Translational Research

Biodistribution and safety assessment of AAV2-GAD following intrasubthalamic injection in the rat

(2010) Helen L. Fitzsimons et al.   JOURNAL OF GENE MEDICINE

The Major Locus for Mouse Adenovirus Susceptibility Maps to Genes of the Hematopoietic Cell Surface-Expressed LY6 Family

(2010) K. R. Spindler et al.   JOURNAL OF IMMUNOLOGY

Expression of human A53T alpha-synuclein in the rat substantia nigra using a novel AAV1/2 vector produces a rapidly evolving pathology with protein aggregation, dystrophic neurite architecture and nigrostriatal degeneration with potential to model the pathology of Parkinson's disease

(2010) James B Koprich et al.   Molecular Neurodegeneration

Fcγ receptors are required for NF-κB signaling, microglial activation and dopaminergic neurodegeneration in an AAV-synuclein mouse model of Parkinson's disease

(2010) Shuwen Cao et al.   Molecular Neurodegeneration

Differential Adeno-Associated Virus Mediated Gene Transfer to Sensory Neurons following Intrathecal Delivery by Direct Lumbar Puncture

(2010) Lucy Vulchanova et al.   Molecular Pain

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

Anterograde Axonal Transport of AAV2-GDNF in Rat Basal Ganglia

(2010) Agnieszka Ciesielska 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

Inhibitors of leucine-rich repeat kinase-2 protect against models of Parkinson's disease

(2010) Byoung Dae Lee et al.   NATURE MEDICINE

Qualitative Imaging of Adeno-Associated Virus Serotype 2–Human Aromatic L-Amino Acid Decarboxylase Gene Therapy in a Phase I Study for the Treatment of Parkinson Disease

(2010) Francisco Valles et al.   NEUROSURGERY

AAV9-mediated erythropoietin gene delivery into the brain protects nigral dopaminergic neurons in a rat model of Parkinson's disease

(2009) Y-Q Xue et al.   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

Recombinant Adeno-Associated Virus Serotype 6 (rAAV2/6)-Mediated Gene Transfer to Nociceptive Neurons through Different Routes of Delivery

(2009) Chris Towne et al.   Molecular Pain

AAV9: a potential blood-brain barrier buster

(2009) Fredric P Manfredsson et al.   MOLECULAR THERAPY

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

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

Multicenter Analysis of Glucocerebrosidase Mutations in Parkinson's Disease

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

Targeted Overexpression of Human α-Synuclein Triggers Microglial Activation and an Adaptive Immune Response in a Mouse Model of Parkinson Disease

(2008) Shaji Theodore et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

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

Human Interleukin-10 Gene Transfer Is Protective in a Rat Model of Parkinson's Disease

(2008) Louisa C Johnston et al.   MOLECULAR THERAPY