A next step in adeno-associated virus-mediated gene therapy for neurological diseases: regulation and targeting

The advent of AAV9 expands applications for brain and spinal cord gene delivery

(2012) Robert D Dayton et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Rapid Transgene Expression in Multiple Precursor Cell Types of Adult Rat Subventricular Zone Mediated by Adeno-Associated Type 1 Vectors

(2012) Olivier Bockstael et al.   HUMAN GENE THERAPY

Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice

(2012) Françoise Piguet et al.   HUMAN GENE THERAPY

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

(2012) Gabriele Mittermeyer et al.   HUMAN GENE THERAPY

Gene Transfer to the CNS Is Efficacious in Immune-primed Mice Harboring Physiologically Relevant Titers of Anti-AAV Antibodies

(2012) Christopher M Treleaven et al.   MOLECULAR THERAPY

Patterns of scAAV Vector Insertion Associated With Oncogenic Events in a Mouse Model for Genotoxicity

(2012) Lucia E Rosas et al.   MOLECULAR THERAPY

Cerebral Infusion of AAV9 Vector-encoding Non-self Proteins Can Elicit Cell-mediated Immune Responses

(2012) Agnieszka Ciesielska et al.   MOLECULAR THERAPY

Advancing neurotrophic factors as treatments for age-related neurodegenerative diseases: developing and demonstrating “clinical proof-of-concept” for AAV-neurturin (CERE-120) in Parkinson's disease

(2012) Raymond T. Bartus et al.   NEUROBIOLOGY OF AGING

Regulated protein expression for in vivo gene therapy for neurological disorders: Progress, strategies, and issues

(2012) Fredric P. Manfredsson et al.   NEUROBIOLOGY OF DISEASE

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

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

Guided delivery of adeno-associated viral vectors into the primate brain

(2011) Ernesto A. Salegio et al.   ADVANCED DRUG DELIVERY REVIEWS

Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents

(2011) Roberto Calcedo et al.   Clinical and Vaccine Immunology

Adeno-Associated Virus (AAV) Vectors in the CNS

(2011) Thomas J. McCown   CURRENT GENE THERAPY

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

(2011) Lluis Samaranch et al.   HUMAN GENE THERAPY

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

Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates

(2011) Steven J Gray et al.   MOLECULAR THERAPY

Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

(2011) Hongwei Zhang et al.   MOLECULAR THERAPY

Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery

(2011) Haiyan Fu et al.   MOLECULAR THERAPY

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

(2011) Dawn E Bowles et al.   MOLECULAR THERAPY

Current prospects for RNA interference-based therapies

(2011) Beverly L. Davidson et al.   NATURE REVIEWS GENETICS

Evaluation of an AAV2-Based Rapamycin-Regulated Glial Cell Line-Derived Neurotrophic Factor (GDNF) Expression Vector System

(2011) Piotr Hadaczek et al.   PLoS One

Improved Tet-responsive promoters with minimized background expression

(2010) Rainer Loew et al.   BMC BIOTECHNOLOGY

A General Chemical Method to Regulate Protein Stability in the Mammalian Central Nervous System

(2010) Mari Iwamoto et al.   CHEMISTRY & BIOLOGY

Lack of humoral immune response to the tetracycline (Tet) activator in rats injected intracranially with Tet-off rAAV vectors

(2010) Y Han et al.   GENE THERAPY

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

Adjusting transgene expression levels in lymphocytes with a set of inducible promoters

(2010) Christina Danke et al.   JOURNAL OF GENE MEDICINE

Mutagenesis of Adeno-Associated Virus Type 2 Capsid Protein VP1 Uncovers New Roles for Basic Amino Acids in Trafficking and Cell-Specific Transduction

(2010) J. S. Johnson et al.   JOURNAL OF VIROLOGY

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

Novel Properties of Tyrosine-mutant AAV2 Vectors in the Mouse Retina

(2010) Hilda Petrs-Silva et al.   MOLECULAR THERAPY

MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression

(2010) Jun Xie 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

In vivo gene regulation using tetracycline-regulatable systems

(2009) Knut Stieger et al.   ADVANCED DRUG DELIVERY REVIEWS

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

Transduction of Nonhuman Primate Brain with Adeno-Associated Virus Serotype 1: Vector Trafficking and Immune Response

(2009) Piotr Hadaczek et al.   HUMAN GENE THERAPY

Human α-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution

(2009) Carine Ciron 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

Engineering adeno-associated virus serotype 2-based targeting vectors using a new insertion site-position 453-and single point mutations

(2009) Jorge Boucas et al.   JOURNAL OF GENE MEDICINE

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Comparison of transduction efficiency of recombinant AAV serotypes 1, 2, 5, and 8 in the rat nigrostriatal system

(2009) Nikolaus R. McFarland et al.   JOURNAL OF NEUROCHEMISTRY

Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

(2009) Sandra Duque et al.   MOLECULAR THERAPY

Efficient Gene Delivery and Selective Transduction of Glial Cells in the Mammalian Brain by AAV Serotypes Isolated From Nonhuman Primates

(2009) Patricia A Lawlor et al.   MOLECULAR THERAPY

Tight Long-term Dynamic Doxycycline Responsive Nigrostriatal GDNF Using a Single rAAV Vector

(2009) Fredric P Manfredsson et al.   MOLECULAR THERAPY

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

Molecular Evolution of Adeno-associated Virus for Enhanced Glial Gene Delivery

(2009) James T Koerber et al.   MOLECULAR THERAPY

Differential Transduction Following Basal Ganglia Administration of Distinct Pseudotyped AAV Capsid Serotypes in Nonhuman Primates

(2009) Hemraj B Dodiya et al.   MOLECULAR THERAPY

Comparative Transduction Efficiency of AAV Vector Serotypes 1–6 in the Substantia Nigra and Striatum of the Primate Brain

(2009) Eleni A Markakis et al.   MOLECULAR THERAPY

Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)

(2009) Steven J Gray et al.   MOLECULAR THERAPY

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

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

A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Müller Cells

(2009) Ryan R. Klimczak et al.   PLoS One

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

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

Expression of transgenes in midbrain dopamine neurons using the tyrosine hydroxylase promoter

(2008) M S Oh et al.   GENE THERAPY

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

Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA

(2008) Stefan Worgall et al.   HUMAN GENE THERAPY

Adeno-associated virus-mediated ILK gene silencing in the rat NAc core

(2008) Qiang Chen et al.   JOURNAL OF NEUROSCIENCE METHODS

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

(2008) D. Grimm et al.   JOURNAL OF VIROLOGY

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

Immunization Against the Transgene but not the TetON Switch Reduces Expression From Gutless Adenoviral Vectors in the Brain

(2008) Weidong Xiong et al.   MOLECULAR THERAPY

Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain

(2008) Cassia N Cearley et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

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

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

AAV8, 9, Rh10, Rh43 Vector Gene Transfer in the Rat Brain: Effects of Serotype, Promoter and Purification Method

(2007) Ronald L Klein et al.   MOLECULAR THERAPY