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Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

Annual review of virology, 2019-09, Vol.6 (1), p.601-621

2019

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A largely random AAV integration profile after LPLD gene therapy

Nature medicine, 2013-07, Vol.19 (7), p.889-891

2013

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Lateral Hypothalamic Area Glutamatergic Neurons and Their Projections to the Lateral Habenula Regulate Feeding and Reward

The Journal of neuroscience, 2016-01, Vol.36 (2), p.302-311

2016

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Viral vectors for therapy of neurologic diseases

Neuropharmacology, 2017-07, Vol.120, p.63-80

2017

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Adenoviral Gene Delivery Can Reprogram Human Fibroblasts to Induced Pluripotent Stem Cells

Stem cells (Dayton, Ohio), 2009-11, Vol.27 (11), p.2667-2674

2009

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The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9–dependent innate immune responses in the liver

Blood, 2011-06, Vol.117 (24), p.6459-6468

2011

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pEAQ: versatile expression vectors for easy and quick transient expression of heterologous proteins in plants

Plant biotechnology journal, 2009-09, Vol.7 (7), p.682-693

2009

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MicroRNA-124 is a subventricular zone neuronal fate determinant

The Journal of neuroscience, 2012-06, Vol.32 (26), p.8879-8889

2012

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Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells

Blood, 2013-03, Vol.121 (12), p.2224-2233

2013

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Evoked Axonal Oxytocin Release in the Central Amygdala Attenuates Fear Response

Neuron (Cambridge, Mass.), 2012-02, Vol.73 (3), p.553-566

2012

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c-Jun Reprograms Schwann Cells of Injured Nerves to Generate a Repair Cell Essential for Regeneration

Neuron (Cambridge, Mass.), 2012-08, Vol.75 (4), p.633-647

2012

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Assessing the potential for AAV vector genotoxicity in a murine model

Blood, 2011-03, Vol.117 (12), p.3311-3319

2011

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Enhancers are major targets for murine leukemia virus vector integration

Journal of virology, 2014-04, Vol.88 (8), p.4504-4513

2014

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Recombinant adeno-associated virus utilizes host cell nuclear import machinery to enter the nucleus

Journal of virology, 2014-04, Vol.88 (8), p.4132-4144

2014

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Resilience to social stress coincides with functional DNA methylation of the Crf gene in adult mice

Nature neuroscience, 2010-11, Vol.13 (11), p.1351-1353

2010

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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

Lancet neurology, 2008-05, Vol.7 (5), p.400-408

2008

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survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype

Virology journal, 2013-03, Vol.10 (1), p.74-74, Article 74

2013

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

Neurobiology of disease, 2012-03, Vol.45 (3), p.939-953

2012

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Transgene expression in target-defined neuron populations mediated by retrograde infection with adeno-associated viral vectors

The Journal of neuroscience, 2013-09, Vol.33 (38), p.15195-15206

2013

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Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice

Nature biomedical engineering, 2021-02, Vol.5 (2), p.144-156

2021

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Adenovirus Serotype 5 Neutralizing Antibodies Target both Hexon and Fiber following Vaccination and Natural Infection

Journal of Virology, 2012, Vol.86 (1), p.625-629

2012

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Venezuelan Equine Encephalitis Virus Replicon Particle Vaccine Protects Nonhuman Primates from Intramuscular and Aerosol Challenge with Ebolavirus

Journal of Virology, 2013-05, Vol.87 (9), p.4952-4964

2013

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Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application

Human gene therapy, 2011-03, Vol.22 (3), p.343-356

2011

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Increasing dopamine D2 receptor expression in the adult nucleus accumbens enhances motivation

Molecular psychiatry, 2013-09, Vol.18 (9), p.1025-1033

2013

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Fertility-regulating Kiss1 neurons arise from hypothalamic POMC-expressing progenitors

The Journal of neuroscience, 2015-04, Vol.35 (14), p.5549-5556

2015

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Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

A largely random AAV integration profile after LPLD gene therapy

Lateral Hypothalamic Area Glutamatergic Neurons and Their Projections to the Lateral Habenula Regulate Feeding and Reward

Viral vectors for therapy of neurologic diseases

Adenoviral Gene Delivery Can Reprogram Human Fibroblasts to Induced Pluripotent Stem Cells

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9–dependent innate immune responses in the liver

pEAQ: versatile expression vectors for easy and quick transient expression of heterologous proteins in plants

MicroRNA-124 is a subventricular zone neuronal fate determinant

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells

Evoked Axonal Oxytocin Release in the Central Amygdala Attenuates Fear Response

c-Jun Reprograms Schwann Cells of Injured Nerves to Generate a Repair Cell Essential for Regeneration

Assessing the potential for AAV vector genotoxicity in a murine model

Enhancers are major targets for murine leukemia virus vector integration

Recombinant adeno-associated virus utilizes host cell nuclear import machinery to enter the nucleus

Resilience to social stress coincides with functional DNA methylation of the Crf gene in adult mice

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

survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype

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

Transgene expression in target-defined neuron populations mediated by retrograde infection with adeno-associated viral vectors

Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice

Adenovirus Serotype 5 Neutralizing Antibodies Target both Hexon and Fiber following Vaccination and Natural Infection

Venezuelan Equine Encephalitis Virus Replicon Particle Vaccine Protects Nonhuman Primates from Intramuscular and Aerosol Challenge with Ebolavirus

Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application

Increasing dopamine D2 receptor expression in the adult nucleus accumbens enhances motivation

Fertility-regulating Kiss1 neurons arise from hypothalamic POMC-expressing progenitors

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