(Q38190041)

30 August 2017

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title

Adeno-associated virus vectors and neurological gene therapy (English)

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30 August 2017

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David S Ojala

1

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30 August 2017

Dominic P Amara

series ordinal

2

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30 August 2017

David V Schaffer

series ordinal

3

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30 August 2017

publication date

20 February 2014

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30 August 2017

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30 August 2017

volume

21

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30 August 2017

issue

1

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30 August 2017

page(s)

84-98

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The AAV vector toolkit: poised at the clinical crossroads

30 August 2017

cites work

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

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Adeno-associated viral vectors for mapping, monitoring, and manipulating neural circuits

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Optimized adeno-associated viral vector-mediated striatal DOPA delivery restores sensorimotor function and prevents dyskinesias in a model of advanced Parkinson's disease

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Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector

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Axon degeneration in Parkinson's disease

21 January 2018

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21 January 2018

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Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain.

21 January 2018

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Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain

21 January 2018

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A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease

21 January 2018

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Continuous DOPA synthesis from a single AAV: dosing and efficacy in models of Parkinson's disease

21 January 2018

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Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

21 January 2018

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Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

21 January 2018

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Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses

21 January 2018

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Gene therapy of the brain in the dog model of Hurler's syndrome.

21 January 2018

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Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain

21 January 2018

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In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous

21 January 2018

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Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9.

21 January 2018

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Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

21 January 2018

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

21 January 2018

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Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes.

21 January 2018

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Directed evolution of adeno-associated virus to an infectious respiratory virus

21 January 2018

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Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

21 January 2018

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Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neurons

21 January 2018

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Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS

21 January 2018

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microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors

21 January 2018

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Gene therapy clinical trials worldwide to 2012 - an update.

21 January 2018

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21 January 2018

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21 January 2018

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

21 January 2018

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21 January 2018

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21 January 2018

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Efficient Retrograde Neuronal Transduction Utilizing Self-complementary AAV1.

21 January 2018

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Systemic delivery of tyrosine-mutant AAV vectors results in robust transduction of neurons in adult mice

21 January 2018

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Non-cell autonomous toxicity in neurodegenerative disorders: ALS and beyond

21 January 2018

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An evolved adeno-associated viral variant enhances gene delivery and gene targeting in neural stem cells

21 January 2018

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A largely random AAV integration profile after LPLD gene therapy

21 January 2018

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Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

21 January 2018

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Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model.

21 January 2018

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Efficient gene therapy-based method for the delivery of therapeutics to primate cortex

21 January 2018

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AAV-mediated gene targeting methods for human cells

21 January 2018

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DNA shuffling of adeno-associated virus yields functionally diverse viral progeny

21 January 2018

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Molecular evolution of adeno-associated virus for enhanced glial gene delivery

21 January 2018

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Construction of diverse adeno-associated viral libraries for directed evolution of enhanced gene delivery vehicles

21 January 2018

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21 January 2018

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Reflux-free cannula for convection-enhanced high-speed delivery of therapeutic agents

21 January 2018

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Designer gene delivery vectors: molecular engineering and evolution of adeno-associated viral vectors for enhanced gene transfer

21 January 2018

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Randomized controlled trial of intraputamenal glial cell line-derived neurotrophic factor infusion in Parkinson disease

21 January 2018

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AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

21 January 2018

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https://api.crossref.org/works/10.1177%2F1073858414521870

Prediction of convection-enhanced drug delivery to the human brain.

21 January 2018

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https://api.crossref.org/works/10.1177%2F1073858414521870

Directed evolution of adeno-associated virus for glioma cell transduction

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Directed evolution of adeno-associated virus yields enhanced gene delivery vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Comparative transduction efficiency of AAV vector serotypes 1-6 in the substantia nigra and striatum of the primate brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Immune responses to AAV in a phase I study for Canavan disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Glybera and the future of gene therapy in the European Union

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Convection-enhanced delivery of AAV-2 combined with heparin increases TK gene transfer in the rat brain.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Research advances in gene therapy approaches for the treatment of amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

The blood-brain barrier: bottleneck in brain drug development

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Role of tyrosine phosphorylation of a cellular protein in adeno-associated virus 2-mediated transgene expression

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Virus-mediated shRNA knockdown of Na(v)1.3 in rat dorsal root ganglion attenuates nerve injury-induced neuropathic pain

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Safety and tolerability of magnetic resonance imaging-guided convection-enhanced delivery of AAV2-hAADC with a novel delivery platform in nonhuman primate striatum

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Molecular engineering of viral gene delivery vehicles

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Molecular mechanisms of pathogenesis of Parkinson's disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Terminal N-linked galactose is the primary receptor for adeno-associated virus 9.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Promoters and serotypes: targeting of adeno-associated virus vectors for gene transfer in the rat central nervous system in vitro and in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

A viral assembly factor promotes AAV2 capsid formation in the nucleolus

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Frameless multimodal image guidance of localized convection-enhanced delivery of therapeutics in the brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Adeno-associated virus vector-mediated transduction in the cat brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Glial promoter selectivity following AAV-delivery to the immature brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Adeno-associated virus serotypes: vector toolkit for human gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Efficient retrograde transport of adeno-associated virus type 8 to spinal cord and dorsal root ganglion after vector delivery in muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1073858414521870

21 January 2018

Identifiers

DOI

10.1177/1073858414521870

1 reference

30 August 2017

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