Translational considerations for CNS gene therapy (Q36739755)

16 August 2017

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title

Translational considerations for CNS gene therapy (English)

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16 August 2017

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Deborah A Ryan

1

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16 August 2017

Howard J Federoff

series ordinal

2

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16 August 2017

language of work or name

English

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publication date

1 March 2007

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Expert Opinion on Biological Therapy

16 August 2017

published in

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16 August 2017

volume

7

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16 August 2017

issue

3

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16 August 2017

page(s)

305-318

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Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy

16 August 2017

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21 January 2018

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Tight control of gene expression in mammalian cells by tetracycline-responsive promoters

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Transcriptional activation by tetracyclines in mammalian cells

21 January 2018

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Penetration of oral doxycycline into the cerebrospinal fluid of patients with latent or neurosyphilis

21 January 2018

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Cell-type-specific and regulatable transgenesis in the adult brain: adenovirus-encoded combined transcriptional targeting and inducible transgene expression

21 January 2018

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21 January 2018

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Lack of an immune response against the tetracycline-dependent transactivator correlates with long-term doxycycline-regulated transgene expression in nonhuman primates after intramuscular injection of recombinant adeno-associated virus

21 January 2018

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Gene transfer of a chimeric trans-activator is immunogenic and results in short-lived transgene expression

21 January 2018

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Immune responses against tetracycline-dependent transactivators affect long-term expression of mouse erythropoietin delivered by a helper-dependent adenoviral vector

21 January 2018

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A humanized system for pharmacologic control of gene expression

21 January 2018

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Pharmacological regulation of protein expression from adeno-associated viral vectors in the eye.

21 January 2018

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Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates.

21 January 2018

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Delivery of a stringent dimerizer-regulated gene expression system in a single retroviral vector

21 January 2018

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Drug inducible transgene expression in brain using a herpes simplex virus vector

21 January 2018

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Viral-mediated temporally controlled dopamine production in a rat model of Parkinson disease

21 January 2018

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Pharmacologic transgene control systems for gene therapy

21 January 2018

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21 January 2018

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Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver

21 January 2018

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Selective and rapid uptake of adeno-associated virus type 2 in brain

21 January 2018

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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

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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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Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy.

21 January 2018

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Adeno-associated virus vectors: activity and applications in the CNS.

21 January 2018

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Herpes simplex virus as a transneuronal tracer

21 January 2018

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Gene transfer to neurons using herpes simplex virus-based vectors

21 January 2018

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The herpes simplex virus 2 kb latency associated transcript (LAT) leader sequence allows efficient expression of downstream proteins which is enhanced in neuronal cells: possible function of LAT ORFs

21 January 2018

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HSV-1 amplicon vectors--simplicity and versatility

21 January 2018

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The herpes simplex virus amplicon: A new eucaryotic defective-virus cloning-amplifying vector

21 January 2018

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Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus

21 January 2018

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Prolonged in vivo gene expression driven by a tyrosine hydroxylase promoter in a defective herpes simplex virus amplicon vector

21 January 2018

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Secreted amyloid beta-protein similar to that in the senile plaques of Alzheimer's disease is increased in vivo by the presenilin 1 and 2 and APP mutations linked to familial Alzheimer's disease

21 January 2018

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Synaptic targeting by Alzheimer's-related amyloid beta oligomers.

21 January 2018

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A specific amyloid-beta protein assembly in the brain impairs memory

21 January 2018

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Cholinergic neuropathology in a mouse model of Alzheimer's disease

21 January 2018

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NGF induction of NGF receptor gene expression and cholinergic neuronal hypertrophy within the basal forebrain of the adult rat.

21 January 2018

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NGF deprivation of adult rat brain results in cholinergic hypofunction and selective impairments in spatial learning

21 January 2018

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Continuous infusion of nerve growth factor prevents basal forebrain neuronal death after fimbria fornix transection

21 January 2018

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SNAP-25 and syntaxin, but not synaptobrevin 2, cooperate in the regulated release of nerve growth factor

21 January 2018

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Characterization of Nerve Growth Factor (NGF) Release from Hippocampal Neurons: Evidence for a Constitutive and an Unconventional Sodium‐dependent Regulated Pathway

21 January 2018

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The neurotrophins BDNF, NT-3, and NGF display distinct patterns of retrograde axonal transport in peripheral and central neurons

21 January 2018

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The aging brain: morphomolecular senescence of cortical circuits

21 January 2018

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Loss of cholinergic phenotype in basal forebrain coincides with cognitive decline in a mouse model of Down's syndrome

21 January 2018

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Nerve growth factor expressed in the medial septum following in vivo gene delivery using a recombinant adeno-associated viral vector protects cholinergic neurons from fimbria-fornix lesion-induced degeneration

21 January 2018

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NGF gene transfer to intrinsic basal forebrain neurons increases cholinergic cell size and protects from age-related, spatial memory deficits in middle-aged rats

21 January 2018

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A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease

21 January 2018

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Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Apolipoprotein E: high-avidity binding to beta-amyloid and increased frequency of type 4 allele in late-onset familial Alzheimer disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Protective effect of apolipoprotein E type 2 allele for late onset Alzheimer disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Lack of apolipoprotein E dramatically reduces amyloid beta-peptide deposition

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Apolipoprotein E isoform-dependent amyloid deposition and neuritic degeneration in a mouse model of Alzheimer's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Apolipoprotein E promotes astrocyte colocalization and degradation of deposited amyloid-beta peptides

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Association of human, rat, and rabbit apolipoprotein E with ?-amyloid

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Gene delivery of human apolipoprotein E alters brain Abeta burden in a mouse model of Alzheimer's disease

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Antibodies against beta-amyloid slow cognitive decline in Alzheimer's disease

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Subacute meningoencephalitis in a subset of patients with AD after Abeta42 immunization

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Neuropathology of human Alzheimer disease after immunization with amyloid-beta peptide: a case report.

21 January 2018

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HSV amplicon-mediated Abeta vaccination in Tg2576 mice: differential antigen-specific immune responses.

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Chronic Parkinsonism in humans due to a product of meperidine-analog synthesis

21 January 2018

1 reference

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Mutation in the alpha-synuclein gene identified in families with Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Ala30Pro mutation in the gene encoding alpha-synuclein in Parkinson's disease

21 January 2018

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The new mutation, E46K, of alpha-synuclein causes Parkinson and Lewy body dementia

21 January 2018

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alpha-Synuclein locus triplication causes Parkinson's disease

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Mutations in LRRK2 cause autosomal-dominant parkinsonism with pleomorphic pathology

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.7.3.305

The ubiquitin pathway in Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Familial Parkinson disease gene product, parkin, is a ubiquitin-protein ligase

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Mutations in the parkin gene cause autosomal recessive juvenile parkinsonism

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Localized striatal delivery of GDNF as a treatment for Parkinson disease

21 January 2018

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https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Dopaminergic neurons protected from degeneration by GDNF gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Aberrant sprouting and downregulation of tyrosine hydroxylase in lesioned nigrostriatal dopamine neurons induced by long-lasting overexpression of glial cell line derived neurotrophic factor in the striatum by lentiviral gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Continuous low-level glial cell line-derived neurotrophic factor delivery using recombinant adeno-associated viral vectors provides neuroprotection and induces behavioral recovery in a primate model of Parkinson's disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain

21 January 2018

1 reference

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Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using rAAV-mediated gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Reversal of dyskinesias in an animal model of Parkinson's disease by continuous L-DOPA delivery using rAAV vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Subthalamic GAD gene therapy in a Parkinson's disease rat model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Lentiviral vector delivery of parkin prevents dopaminergic degeneration in an alpha-synuclein rat model of Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Parkin gene therapy for alpha-synucleinopathy: a rat model of Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

Adenoviral-mediated, high-level, cell-specific transgene expression: a SYN1-WPRE cassette mediates increased transgene expression with no loss of neuron specificity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.7.3.305

21 January 2018

Identifiers

DOI

10.1517/14712598.7.3.305

1 reference

16 August 2017

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