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Title
Gene Therapy Tools for Brain Diseases
Authors
Keywords
-
Journal
Frontiers in Pharmacology
Volume 10, Issue -, Pages -
Publisher
Frontiers Media SA
Online
2019-07-01
DOI
10.3389/fphar.2019.00724
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Note: Only part of the references are listed.- Targeting Huntingtin Expression in Patients with Huntington’s Disease
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- (2018) Fang Han et al. JOURNAL OF VIROLOGY
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- (2018) Andrea Maddalena et al. MOLECULAR THERAPY
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- (2018) David Adams et al. NEW ENGLAND JOURNAL OF MEDICINE
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- (2018) Annasara Lenman et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
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- (2018) Magalie Penaud-Budloo et al. Molecular Therapy-Methods & Clinical Development
- Long-Term Follow-Up of a Phase I/II Study of ProSavin, a Lentiviral Vector Gene Therapy for Parkinson's Disease
- (2018) Stéphane Palfi et al. Human Gene Therapy Clinical Development
- Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques
- (2018) Florie Borel et al. Science Translational Medicine
- Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems
- (2017) Ken Y Chan et al. NATURE NEUROSCIENCE
- Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
- (2017) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Cellular selectivity of AAV serotypes for gene delivery in neurons and astrocytes by neonatal intracerebroventricular injection
- (2017) Sean L. Hammond et al. PLoS One
- Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy
- (2017) Sarah L. DeVos et al. Science Translational Medicine
- Altering neuronal excitability to preserve network connectivity in a computational model of Alzheimer's disease
- (2017) Willem de Haan et al. PLoS Computational Biology
- Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin
- (2017) Edith L. Pfister et al. Molecular Therapy-Nucleic Acids
- Deletion of the Virion Host Shut-off Gene Enhances Neuronal-Selective Transgene Expression from an HSV Vector Lacking Functional IE Genes
- (2017) Yoshitaka Miyagawa et al. Molecular Therapy-Methods & Clinical Development
- Engineered HSV vector achieves safe long-term transgene expression in the central nervous system
- (2017) Gianluca Verlengia et al. Scientific Reports
- Single point mutation in adeno-associated viral vectors -DJ capsid leads to improvement for gene delivery in vivo
- (2016) Yingying Mao et al. BMC BIOTECHNOLOGY
- A brain microvasculature endothelial cell‐specific viral vector with the potential to treat neurovascular and neurological diseases
- (2016) Jakob Körbelin et al. EMBO Molecular Medicine
- Current status of non-viral gene therapy for CNS disorders
- (2016) Rahul Dev Jayant et al. Expert Opinion on Drug Delivery
- Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector
- (2016) Sourav R Choudhury et al. MOLECULAR THERAPY
- A thalamic input to the nucleus accumbens mediates opiate dependence
- (2016) Yingjie Zhu et al. NATURE
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- (2016) Benjamin E Deverman et al. NATURE BIOTECHNOLOGY
- PPARγ-coactivator-1α gene transfer reduces neuronal loss and amyloid-β generation by reducing β-secretase in an Alzheimer’s disease model
- (2016) Loukia Katsouri et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- 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
- Genome-Editing Technologies: Principles and Applications
- (2016) Thomas Gaj et al. Cold Spring Harbor Perspectives in Biology
- Systemic delivery of adeno-associated viral vectors
- (2016) Dongsheng Duan Current Opinion in Virology
- Recombinant Human Myelin-Associated Glycoprotein Promoter Drives Selective AAV-Mediated Transgene Expression in Oligodendrocytes
- (2016) Georg von Jonquieres et al. Frontiers in Molecular Neuroscience
- Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease
- (2016) Jana Miniarikova et al. Molecular Therapy-Nucleic Acids
- Microglia-specific targeting by novel capsid-modified AAV6 vectors
- (2016) Awilda M Rosario et al. Molecular Therapy-Methods & Clinical Development
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- (2016) Chiara Falcicchia et al. PLoS One
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- (2015) Richard S. Geary et al. ADVANCED DRUG DELIVERY REVIEWS
- Patterned, But Not Tonic, Optogenetic Stimulation in Motor Thalamus Improves Reaching in Acute Drug-Induced Parkinsonian Rats
- (2015) S. Seeger-Armbruster et al. JOURNAL OF NEUROSCIENCE
- 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
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- (2014) Mathieu Bourdenx et al. Frontiers in Molecular Neuroscience
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- (2013) Kevin D Foust et al. MOLECULAR THERAPY
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- (2013) Michele Simonato et al. Nature Reviews Neurology
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- (2013) Georg von Jonquieres et al. PLoS One
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- (2013) Dominik F. Aschauer et al. PLoS One
- On-demand optogenetic control of spontaneous seizures in temporal lobe epilepsy
- (2013) Esther Krook-Magnuson et al. Nature Communications
- Kinetics of rapamycin production by Streptomyces hygroscopicus MTCC 4003
- (2013) Subhasish Dutta et al. 3 Biotech
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- (2011) Hongwei Zhang et al. MOLECULAR THERAPY
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- (2011) Francisco C. Pérez-Martínez et al. PHARMACEUTICAL RESEARCH
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- (2011) Piotr Hadaczek et al. PLoS One
- Chemical and Genetic Engineering of Selective Ion Channel-Ligand Interactions
- (2011) C. J. Magnus et al. SCIENCE
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- (2010) Michael R. Lieber Annual Review of Biochemistry
- Epigenetic regulation of latent HSV-1 gene expression
- (2010) David C. Bloom et al. Biochimica et Biophysica Acta-Gene Regulatory Mechanisms
- Penetration of Drugs through the Blood-Cerebrospinal Fluid/Blood-Brain Barrier for Treatment of Central Nervous System Infections
- (2010) R. Nau et al. CLINICAL MICROBIOLOGY REVIEWS
- Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations
- (2010) Mark M. Souweidane et al. Journal of Neurosurgery-Pediatrics
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- (2010) Fang Zhang et al. MOLECULAR THERAPY
- High-performance genetically targetable optical neural silencing by light-driven proton pumps
- (2010) Brian Y. Chow et al. NATURE
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- (2010) Alexxai V. Kravitz et al. NATURE
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- (2010) Karl Deisseroth NATURE METHODS
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- (2010) Lisa A Gunaydin et al. NATURE NEUROSCIENCE
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- (2010) Jesse R. Raab et al. NATURE REVIEWS GENETICS
- Systematic Comparison of Constitutive Promoters and the Doxycycline-Inducible Promoter
- (2010) Jane Yuxia Qin et al. PLoS One
- “Armed” oncolytic herpes simplex viruses for brain tumor therapy
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- Sustained effects of nonallele-specificHuntingtinsilencing
- (2009) Valérie Drouet et al. ANNALS OF NEUROLOGY
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- An Improved Tet-On System for Gene Expression in Neurons Delivered by a Single Lentiviral Vector
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- Cortical hyperexcitability and epileptogenesis: Understanding the mechanisms of epilepsy - Part 2
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- Intranuclear fluorescence resonance energy transfer analysis of plasmid DNA decondensation from nonviral gene carriers
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- HSV-1-derived amplicon vectors: recent technological improvements and remaining difficulties - a review
- (2009) Alberto Luis Epstein MEMORIAS DO INSTITUTO OSWALDO CRUZ
- Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice
- (2009) Ryan L Boudreau et al. MOLECULAR THERAPY
- Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy
- (2009) Yong Hong Chen et al. NATURE MEDICINE
- Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
- (2009) C. W. Christine et al. NEUROLOGY
- Remote Control of Neuronal Activity in Transgenic Mice Expressing Evolved G Protein-Coupled Receptors
- (2009) Georgia M. Alexander et al. NEURON
- Dopamine Gene Therapy for Parkinson's Disease in a Nonhuman Primate Without Associated Dyskinesia
- (2009) B. Jarraya et al. Science Translational Medicine
- Gene therapy: the end of the rainbow?
- (2009) Edward J Shillitoe Head and Neck Optical Diagnostics Society
- Viral vectors: from virology to transgene expression
- (2008) D Bouard et al. BRITISH JOURNAL OF PHARMACOLOGY
- Homologous recombination in DNA repair and DNA damage tolerance
- (2008) Xuan Li et al. CELL RESEARCH
- GFAPpromoter elements required for region-specific and astrocyte-specific expression
- (2008) Youngjin Lee et al. GLIA
- Lentiviral Vectors Mediate Nonimmunosuppressive Rapamycin Analog-Induced Production of Secreted Therapeutic Factors in the Brain: Regulation at the Level of Transcription and Exocytosis
- (2008) Roland Vogel et al. HUMAN GENE THERAPY
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- (2008) Stefan Worgall et al. HUMAN GENE THERAPY
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- (2008) M. Piest et al. JOURNAL OF CONTROLLED RELEASE
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- (2008) D. Grimm et al. JOURNAL OF VIROLOGY
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- (2008) J. L. Eberling et al. NEUROLOGY
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- (2008) Cong Bao Kang et al. NEUROSIGNALS
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- (2008) Julia Höck et al. GENOME BIOLOGY
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- (2007) Ina Gruh et al. JOURNAL OF GENE MEDICINE
- A Hybrid Vector System Expands Adeno-associated Viral Vector Packaging Capacity in a Transgene-independent Manner
- (2007) Arkasubhra Ghosh et al. MOLECULAR THERAPY
- Breaking the Bonds: Non-viral Vectors Become Chemically Dynamic
- (2007) Jon A Wolff et al. MOLECULAR THERAPY
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