Viral vectors for neurotrophic factor delivery: a gene therapy approach for neurodegenerative diseases of the CNS

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Viral vectors for neurotrophic factor delivery: a gene therapy approach for neurodegenerative diseases of the CNS is …
instance of (P31):
review articleQ7318358
scholarly articleQ13442814

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P356DOI10.1016/J.PHRS.2009.10.002
P8608Fatcat IDrelease_tvyjraogwnhihiazxmpy4iiffq
P932PMC publication ID2880921
P698PubMed publication ID19840853
P5875ResearchGate publication ID38021906

P50authorMikko AiravaaraQ42635702
Brandon K. HarveyQ64867341
P2093author name stringSeung T Lim
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Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirusQ24523073
Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry stepsQ24533013
Adeno-associated virus vector integrationQ24624120
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The GDNF family: signalling, biological functions and therapeutic valueQ28216478
Neurturin shares receptors and signal transduction pathways with glial cell line-derived neurotrophic factor in sympathetic neuronsQ28241378
HSV vector-mediated modification of primary nociceptor afferents: an approach to inhibit chronic painQ37439341
Long-term functional recovery from age-induced spatial memory impairments by nerve growth factor gene transfer to the rat basal forebrainQ37517550
A NERVE GROWTH-STIMULATING FACTOR ISOLATED FROM SARCOM AS 37 AND 180.Q37687787
Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes.Q39596516
Heparan sulfate glycosaminoglycans are receptors sufficient to mediate the initial binding of adenovirus types 2 and 5.Q39607095
Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deletedQ39642143
The nerve growth factor 35 years laterQ39667849
A novel gene expression control system and its use in stable, high-titer 293 cell-based adeno-associated virus packaging cell linesQ39685433
Expression, bioactivity, and safety 1 year after adeno-associated viral vector type 2-mediated delivery of neurturin to the monkey nigrostriatal system support cere-120 for Parkinson's diseaseQ39863620
Adenovirus type 11 uses CD46 as a cellular receptorQ39864028
Construction of adenovirus vectors through Cre-lox recombinationQ39878424
Cytotoxicity of a replication-defective mutant of herpes simplex virus type 1Q40065369
MPTP-induced deficits in striatal synaptic plasticity are prevented by glial cell line-derived neurotrophic factor expressed via an adeno-associated viral vector.Q40095787
Seizure suppression by GDNF gene therapy in animal models of epilepsyQ40154016
Striatal delivery of neurturin by CERE-120, an AAV2 vector for the treatment of dopaminergic neuron degeneration in Parkinson's diseaseQ40197232
Neuroprotection of adenoviral-vector-mediated GDNF expression against kainic-acid-induced excitotoxicity in the rat hippocampusQ40281192
Human neural progenitors deliver glial cell line-derived neurotrophic factor to parkinsonian rodents and aged primatesQ40339968
Lentivector-mediated delivery of GDNF protects complex motor functions relevant to human Parkinsonism in a rat lesion model.Q40347828
Functional reinnervation from remaining DA terminals induced by GDNF lentivirus in a rat model of early Parkinson's disease.Q40387759
Neurturin exerts potent actions on survival and function of midbrain dopaminergic neuronsQ28274311
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AAV2-mediated delivery of human neurturin to the rat nigrostriatal system: long-term efficacy and tolerability of CERE-120 for Parkinson's diseaseQ28304068
Corticostriatopallidal neuroprotection by adenovirus-mediated ciliary neurotrophic factor gene transfer in a rat model of progressive striatal degenerationQ28581577
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Multiply attenuated lentiviral vector achieves efficient gene delivery in vivoQ29616612
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Expanded-capacity adenoviral vectors--the helper-dependent vectorsQ33544035
A replication-incompetent adenovirus vector with the preterminal protein gene deleted efficiently transduces mouse earsQ33640285
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 ratsQ33745707
In vitro and in vivo biology of recombinant adenovirus vectors with E1, E1/E2A, or E1/E4 deleted.Q33782356
Persistence and expression of the herpes simplex virus genome in the absence of immediate-early proteinsQ33783411
Self-inactivating lentivirus vector for safe and efficient in vivo gene deliveryQ33786060
AAV2-mediated gene transfer of GDNF to the striatum of MPTP monkeys enhances the survival and outgrowth of co-implanted fetal dopamine neurons.Q33791830
Failed retrograde transport of NGF in a mouse model of Down's syndrome: reversal of cholinergic neurodegenerative phenotypes following NGF infusionQ33943282
Nontropic actions of neurotrophins: subcortical nerve growth factor gene delivery reverses age-related degeneration of primate cortical cholinergic innervationQ34113268
Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivoQ34277742
Nerve growth factor in Alzheimer's disease: increased levels throughout the brain coupled with declines in nucleus basalisQ34304615
Bridging grafts and transient nerve growth factor infusions promote long-term central nervous system neuronal rescue and partial functional recoveryQ34351483
Transduction profiles of recombinant adeno-associated virus vectors derived from serotypes 2 and 5 in the nigrostriatal system of ratsQ34436780
Adeno-associated virus vectors can be efficiently produced without helper virusQ34479933
AAV hybrid serotypes: improved vectors for gene deliveryQ34619573
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trialQ34640765
Results from a phase I safety trial of hAADC gene therapy for Parkinson diseaseQ34656075
Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain.Q34664932
Viral delivery of glial cell line-derived neurotrophic factor improves behavior and protects striatal neurons in a mouse model of Huntington's diseaseQ34695123
Development and applications of non-HIV-based lentiviral vectors in neurological disordersQ34902118
Immune responses to replication-defective HSV-1 type vectors within the CNS: implications for gene therapyQ35131218
Peripheral infection with adenovirus causes unexpected long-term brain inflammation in animals injected intracranially with first-generation, but not with high-capacity, adenovirus vectors: toward realistic long-term neurological gene therapy for chQ35161530
Lentiviral vectors for treating and modeling human CNS disordersQ35880080
Update on herpesvirus amplicon vectorsQ35903203
Promoters and serotypes: targeting of adeno-associated virus vectors for gene transfer in the rat central nervous system in vitro and in vivoQ35948485
???Q64821040
Long-term glial cell line-derived neurotrophic factor overexpression in the intact nigrostriatal system in rats leads to a decrease of dopamine and increase of tetrahydrobiopterin productionQ40413793
Effect of AdGDNF on dopaminergic neurotransmission in the striatum of 6-OHDA-treated ratsQ40427208
Delayed gene therapy of glial cell line-derived neurotrophic factor is efficacious in a rat model of Parkinson's diseaseQ40442685
Neuroprotective gene therapy for Huntington's disease, using polymer-encapsulated cells engineered to secrete human ciliary neurotrophic factor: results of a phase I study.Q40462605
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.Q40464992
Overexpression of glial cell line-derived neurotrophic factor using a lentiviral vector induces time- and dose-dependent downregulation of tyrosine hydroxylase in the intact nigrostriatal dopamine system.Q40532718
AAV-mediated gene delivery of BDNF or GDNF is neuroprotective in a model of Huntington diseaseQ40560840
Neuroprotection in a rat Parkinson model by GDNF gene therapy using EIAV vectorQ40568114
Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV envelope proteinsQ40583034
Recombinant adeno-associated viral vector (rAAV) delivery of GDNF provides protection against 6-OHDA lesion in the common marmoset monkey (Callithrix jacchus).Q40613492
Protective effects of intracerebral adenoviral-mediated GDNF gene transfer in a rat model of Parkinson's diseaseQ40633580
HSV amplicon delivery of glial cell line-derived neurotrophic factor is neuroprotective against ischemic injury.Q40637488
Structural and functional neuroprotection in a rat model of Huntington's disease by viral gene transfer of GDNF.Q40643169
Long-term striatal overexpression of GDNF selectively downregulates tyrosine hydroxylase in the intact nigrostriatal dopamine systemQ40675429
Glial cell line-derived neurotrophic factor modulates kindling and activation-induced sprouting in hippocampus of adult ratsQ40686200
Insect cells as a factory to produce adeno-associated virus type 2 vectorsQ40690831
Neuroprotective effects of glial cell line-derived neurotrophic factor mediated by an adeno-associated virus vector in a transgenic animal model of amyotrophic lateral sclerosis.Q40710699
Lentivirally delivered glial cell line-derived neurotrophic factor increases the number of striatal dopaminergic neurons in primate models of nigrostriatal degeneration.Q40723341
Adeno-associated viral delivery of GDNF promotes recovery of dopaminergic phenotype following a unilateral 6-hydroxydopamine lesion.Q40723600
Delayed delivery of AAV-GDNF prevents nigral neurodegeneration and promotes functional recovery in a rat model of Parkinson's diseaseQ40737427
Adenovirus-mediated glial cell line-derived neurotrophic factor gene delivery reduces motor neuron injury after transient spinal cord ischemia in rabbitsQ40737759
Neuroprotection in the rat Parkinson model by intrastriatal GDNF gene transfer using a lentiviral vectorQ40741282
Systematic determination of the packaging limit of lentiviral vectorsQ40776442
Bcl-2 and GDNF delivered by HSV-mediated gene transfer act additively to protect dopaminergic neurons from 6-OHDA-induced degenerationQ40805293
Glial cell line-derived neurotrophic factor (GDNF) gene delivery protects dopaminergic terminals from degenerationQ40811477
Rescue of ischemic brain injury by adenoviral gene transfer of glial cell line-derived neurotrophic factor after transient global ischemia in gerbilsQ40839243
Recombinant adeno-associated virus vector expressing glial cell line-derived neurotrophic factor reduces ischemia-induced damageQ40841418
Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's diseaseQ40846012
Pharmacologic treatment of cognition in Alzheimer's dementiaQ40850299
Lentiviral vectors as a gene delivery system in the mouse midbrain: cellular and behavioral improvements in a 6-OHDA model of Parkinson's disease using GDNF.Q40870371
Long-term rAAV-mediated gene transfer of GDNF in the rat Parkinson's model: intrastriatal but not intranigral transduction promotes functional regeneration in the lesioned nigrostriatal system.Q40874496
Self-inactivating lentiviral vectors with enhanced transgene expression as potential gene transfer system in Parkinson's diseaseQ40904090
Differential effects of glial cell line-derived neurotrophic factor (GDNF) in the striatum and substantia nigra of the aged Parkinsonian rat.Q40905804
Intramuscular grafts of myoblasts genetically modified to secrete glial cell line-derived neurotrophic factor prevent motoneuron loss and disease progression in a mouse model of familial amyotrophic lateral sclerosisQ40935559
Behavioral and cellular protection of rat dopaminergic neurons by an adenoviral vector encoding glial cell line-derived neurotrophic factor.Q40983409
Adenovirus-mediated transduction with human glial cell line-derived neurotrophic factor gene prevents 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine-induced dopamine depletion in striatum of mouse brainQ41090635
Dopaminergic neurons protected from degeneration by GDNF gene therapyQ41128316
Role of T cells in inflammation caused by adenovirus vectors in the brain.Q41187652
Nerve growth factor and the neurotrophic factor hypothesisQ41188098
Implants of polymer-encapsulated human NGF-secreting cells in the nonhuman primate: rescue and sprouting of degenerating cholinergic basal forebrain neuronsQ41433192
Nerve growth factor treatment after brain injury prevents neuronal deathQ41478262
Potential use of herpes simplex virus (HSV) vectors for gene therapy of neurological disordersQ41543685
Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brainQ42280576
Therapeutic potential of CERE-110 (AAV2-NGF): targeted, stable, and sustained NGF delivery and trophic activity on rodent basal forebrain cholinergic neuronsQ42435326
Trophic factor effects on septal cholinergic neurons.Q42469153
Regulated lentiviral NGF gene transfer controls rescue of medial septal cholinergic neuronsQ42478941
Nerve growth factor in Alzheimerʼs disease: defective retrograde transport to nucleus basalisQ42481313
Somatic gene transfer of NGF to the aged brain: behavioral and morphological ameliorationQ42485030
NGF gene transfer to intrinsic basal forebrain neurons increases cholinergic cell size and protects from age-related, spatial memory deficits in middle-aged ratsQ42492745
Nerve growth factor reverses neuronal atrophy in a Down syndrome model of age-related neurodegenerationQ42503374
Distribution of [125I]nerve growth factor in the rat brain following a single intraventricular injection: correlation with the topographical distribution of trkA messenger RNA-expressing cellsQ42505941
Gene therapy in the adult primate brain: intraparenchymal grafts of cells genetically modified to produce nerve growth factor prevent cholinergic neuronal degeneration.Q42520774
Nerve growth factor promotes survival of septal cholinergic neurons after fimbrial transectionsQ42525515
Adenovirus type 5 fiber knob binds to MHC class I alpha2 domain at the surface of human epithelial and B lymphoblastoid cellsQ42616240
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 trialQ42650874
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytesQ42739394
AAV-BDNF mediated attenuation of quinolinic acid-induced neuropathology and motor function impairmentQ43704735
Grafting genetically modified cells to the damaged brain: restorative effects of NGF expressionQ43839045
Dose-dependent neuroprotective effect of ciliary neurotrophic factor delivered via tetracycline-regulated lentiviral vectors in the quinolinic acid rat model of Huntington's disease.Q44213139
Transgene expression, bioactivity, and safety of CERE-120 (AAV2-neurturin) following delivery to the monkey striatumQ44631221
Brain-derived neurotrophic factor-mediated protection of striatal neurons in an excitotoxic rat model of Huntington's disease, as demonstrated by adenoviral gene transferQ44694208
Immunological instability of persistent adenovirus vectors in the brain: peripheral exposure to vector leads to renewed inflammation, reduced gene expression, and demyelinationQ44907561
Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous systemQ45007196
Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors.Q45245677
Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's diseaseQ45292300
Neurturin gene therapy improves motor function and prevents death of striatal neurons in a 3-nitropropionic acid rat model of Huntington's diseaseQ45304363
Intraspinal cord delivery of IGF-I mediated by adeno-associated virus 2 is neuroprotective in a rat model of familial ALS.Q45387438
Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brainQ45420843
Stroke and TGF-beta proteins: glial cell line-derived neurotrophic factor and bone morphogenetic protein.Q36020135
Viral vector strategy for glial cell line-derived neurotrophic factor therapy for strokeQ36251160
Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's diseaseQ36288717
Lentivirus-mediated gene transfer to the central nervous system: therapeutic and research applicationsQ36366788
Adenoviral vectors--how to use them in cancer gene therapy?Q36419822
Intraparenchymal spinal cord delivery of adeno-associated virus IGF-1 is protective in the SOD1G93A model of ALSQ36491424
Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cellsQ36506251
Delivery of large genomic DNA inserts >100 kb using HSV-1 amplicons.Q36512694
Tropism and toxicity of adeno-associated viral vector serotypes 1, 2, 5, 6, 7, 8, and 9 in rat neurons and glia in vitro.Q36541150
Intrastriatal injection of an adenoviral vector expressing glial-cell-line-derived neurotrophic factor prevents dopaminergic neuron degeneration and behavioral impairment in a rat model of Parkinson disease.Q36561684
Neurotrophic factors and amyotrophic lateral sclerosisQ36565607
Angels and demons: neurotrophic factors and epilepsy.Q36630307
Neurotrophic signaling cascades in the pathophysiology and treatment of bipolar disorderQ36630501
Transduction of brain by herpes simplex virus vectorsQ36681937
Production and characterization of adeno-associated viral vectorsQ36779416
Use of nonintegrating lentiviral vectors for gene therapyQ36831097
Nerve growth factor gene therapy in Alzheimer diseaseQ36838863
Comparison of the capability of GDNF, BDNF, or both, to protect nigrostriatal neurons in a rat model of Parkinson's diseaseQ36969309
Long-term reversal of cholinergic neuronal decline in aged non-human primates by lentiviral NGF gene deliveryQ37076735
Issues regarding gene therapy products for Parkinson's disease: the development of CERE-120 (AAV-NTN) as one reference pointQ37082159
The biology of neurotrophins, signalling pathways, and functional peptide mimetics of neurotrophins and their receptorsQ37090137
Ex vivo and in vivo gene delivery to the brainQ37144385
A human trial of HSV-mediated gene transfer for the treatment of chronic painQ37195741
GDNF therapy for Parkinson's diseaseQ37203371
Cellular immune response to cryptic epitopes during therapeutic gene transferQ37250034
Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vectorQ37251114
Development and evaluation of a novel gene delivery vehicle composed of adenovirus serotype 35.Q37283186
Functional effects of AAV2-GDNF on the dopaminergic nigrostriatal pathway in parkinsonian rhesus monkeysQ37301013
Real-time MR imaging of adeno-associated viral vector delivery to the primate brainQ37313954
Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activityQ37331730
Non-integrating lentiviral vectors.Q37347158
Clinical application of lentiviral vectors - concepts and practiceQ37347167
Clinically relevant effects of convection-enhanced delivery of AAV2-GDNF on the dopaminergic nigrostriatal pathway in aged rhesus monkeysQ37399627
Continuous infusion of nerve growth factor prevents basal forebrain neuronal death after fimbria fornix transectionQ37410352
Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivoQ45709105
Selective gene expression in brain microglia mediated via adeno-associated virus type 2 and type 5 vectorsQ45727079
Deletion of multiple immediate-early genes from herpes simplex virus reduces cytotoxicity and permits long-term gene expression in neuronsQ45751217
Virus isolation from and identification of HTLV-III/LAV-producing cells in brain tissue from a patient with AIDS.Q45831294
Adenovirus serotype 35 vector-mediated transduction into human CD46-transgenic miceQ45856784
Improvement of spatial learning and memory after adenovirus-mediated transfer of the nerve growth factor gene to aged rat brainQ45858011
Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brainQ45866495
CD46 is a cellular receptor for group B adenovirusesQ45869560
Acute direct adenoviral vector cytotoxicity and chronic, but not acute, inflammatory responses correlate with decreased vector-mediated transgene expression in the brainQ45874048
Gene therapy of murine motor neuron disease using adenoviral vectors for neurotrophic factorsQ45881242
A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer diseaseQ45883058
Intracerebroventricular infusion of nerve growth factor in three patients with Alzheimer's diseaseQ47695139
Changes in brain cholinesterases in senile dementia of Alzheimer typeQ48176243
Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeysQ48197305
Amelioration of cholinergic neuron atrophy and spatial memory impairment in aged rats by nerve growth factorQ48208108
Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeysQ48325968
Continuous infusion of neurotrophin-3 triggers sprouting, decreases the levels of TrkA and TrkC, and inhibits epileptogenesis and activity-dependent axonal growth in adult ratsQ48437842
Nerve growth factor affects 11C-nicotine binding, blood flow, EEG, and verbal episodic memory in an Alzheimer patient (case report).Q48570524
Exogenous NGF affects cholinergic transmitter function and Y-maze behavior in aged Fischer 344 male ratsQ48677998
Specific tropism of HIV-1 for microglial cells in primary human brain culturesQ48920198
Correlation of cholinergic abnormalities with senile plaques and mental test scores in senile dementia.Q50869517
Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model.Q51659718
Human nerve growth factor improves spatial memory in aged but not in young rats.Q52057103
Update on adenovirus and its vectorsQ64380348
Effect of recombinant human insulin-like growth factor-I on progression of ALS. A placebo-controlled study. The North America ALS/IGF-I Study GroupQ73966063
Selective growth stimulating effects of mouse sarcoma on the sensory and sympathetic nervous system of the chick embryoQ75702642
A placebo-controlled trial of insulin-like growth factor-I in amyotrophic lateral sclerosis. European ALS/IGF-I Study GroupQ77114422
A controlled trial of recombinant methionyl human BDNF in ALS: The BDNF Study Group (Phase III)Q77398255
Oncolytic virus therapy using genetically engineered herpes simplex virusesQ81549503
P433issue1
P921main subjectvector-borne diseaseQ2083837
gene therapyQ213901
neurodegenerationQ1755122
P304page(s)14-26
P577publication date2009-10-17
P1433published inPharmacological ResearchQ15724622
P1476titleViral vectors for neurotrophic factor delivery: a gene therapy approach for neurodegenerative diseases of the CNS
P478volume61

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