(Q37687420)

22 August 2017

1 reference

title

Viral vector-mediated gene transfer for CNS disease (English)

1 reference

22 August 2017

0 references

Brooke R Snyder

1

1 reference

22 August 2017

Nicholas M Boulis

series ordinal

2

1 reference

22 August 2017

Thais Federici

series ordinal

3

1 reference

22 August 2017

language of work or name

English

0 references

publication date

1 March 2010

1 reference

Expert Opinion on Biological Therapy

22 August 2017

published in

1 reference

22 August 2017

volume

10

1 reference

22 August 2017

issue

3

1 reference

22 August 2017

page(s)

381-394

1 reference

Molecular Structure of Nucleic Acids: A Structure for Deoxyribose Nucleic Acid

22 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Gene therapy for human genetic disease?

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

The prospects for gene therapy in humans

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

The use of DNA for gene therapy--the need, experimental approach, and implications

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

The future for gene therapy--a reevaluation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Clinical trials with retrovirus mediated gene therapy--what have we learned?

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Development of inducible EIAV-based lentiviral vector packaging and producer cell lines

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Production, concentration and titration of pseudotyped HIV-1-based lentiviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Development and applications of non-HIV-based lentiviral vectors in neurological disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Applications of lentiviral vectors for biology and gene therapy of neurological disorders.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Altering the tropism of lentiviral vectors through pseudotyping

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Robust in vivo gene transfer into adult mammalian neural stem cells by lentiviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Lentiviral vector-mediated genetic modification of human neural progenitor cells for ex vivo gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

HSV1716 injection into the brain adjacent to tumour following surgical resection of high-grade glioma: safety data and long-term survival

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

A human trial of HSV-mediated gene transfer for the treatment of chronic pain

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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 ch

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

One-year expression from high-capacity adenoviral vectors in the brains of animals with pre-existing anti-adenoviral immunity: clinical implications.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Adenoviral virotherapy for malignant brain tumors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

AdvHSV-tk gene therapy with intravenous ganciclovir improves survival in human malignant glioma: a randomised, controlled study

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Immune responses to gene therapy vectors: influence on vector function and effector mechanisms

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Reporter expression persists 1 year after adeno-associated virus-mediated gene transfer to the optic nerve

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Adeno-associated virus (AAV) vectors in the CNS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Clinical gene therapy using recombinant adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Comparison of transduction efficiency of recombinant AAV serotypes 1, 2, 5, and 8 in the rat nigrostriatal system

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Adeno-associated virus type 4 (AAV4) targets ependyma and astrocytes in the subventricular zone and RMS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Recombinant AAV-mediated gene delivery to the central nervous system.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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 system

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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/10.1517%2F14712590903514074

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Preclinical safety validation of a stabilized viral vector direct injection approach to the cervical spinal cord

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Targeted spinal cord therapeutics delivery: stabilized platform and microelectrode recording guidance validation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Cervical spinal cord therapeutics delivery: preclinical safety validation of a stabilized microinjection platform

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Transgene expression, bioactivity, and safety of CERE-120 (AAV2-neurturin) following delivery to the monkey striatum

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Efficient Retrograde Neuronal Transduction Utilizing Self-complementary AAV1.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Gene therapy for the treatment of sensory neuropathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Global diffuse distribution in the brain and efficient gene delivery to the dorsal root ganglia by intrathecal injection of adeno-associated viral vector serotype 1.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Recombinant adeno-associated virus serotype 6 (rAAV2/6)-mediated gene transfer to nociceptive neurons through different routes of delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Sensory neuron targeting by self-complementary AAV8 via lumbar puncture for chronic pain

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

A paracrine paradigm for in vivo gene therapy in the central nervous system: treatment of chronic pain.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Delivery to the central nervous system of a nonreplicative herpes simplex type 1 vector engineered with the interleukin 4 gene protects rhesus monkeys from hyperacute autoimmune encephalomyelitis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Safety of direct administration of AAV2(CU)hCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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/10.1517%2F14712590903514074

Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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%2F14712590903514074

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Long-term reversal of cholinergic neuronal decline in aged non-human primates by lentiviral NGF gene delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Therapeutic potential of CERE-110 (AAV2-NGF): targeted, stable, and sustained NGF delivery and trophic activity on rodent basal forebrain cholinergic neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Regulatory approvals of pediatric oncology drugs: previous experience and new initiatives

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Treatment of advanced CNS malignancies with the recombinant adenovirus H5.010RSVTK: a phase I trial.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Phase I study of adenoviral delivery of the HSV-tk gene and ganciclovir administration in patients with current malignant brain tumors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Adenovirus/herpes simplex-thymidine kinase/ganciclovir complex: preliminary results of a phase I trial in patients with recurrent malignant gliomas.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Ark floats gene therapy's boat, for now.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

A preclinical assessment of the safety and biodistribution of an adenoviral vector containing the herpes simplex virus thymidine kinase gene (Cerepro) after intracerebral administration

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

A Phase I Trial of Ad.hIFN-β Gene Therapy for Glioma

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Neurturin gene therapy improves motor function and prevents death of striatal neurons in a 3-nitropropionic acid rat model of Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Anticonvulsant and antiepileptogenic effects mediated by adeno-associated virus vector neuropeptide Y expression in the rat hippocampus.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Adeno-associated virus-mediated expression and constitutive secretion of galanin suppresses limbic seizure activity in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Neurological manifestations in lysosomal storage disorders - from pathology to first therapeutic possibilities

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

CNS-directed gene therapy for lysosomal storage diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Gene therapy for lysosomal storage diseases (LSDs) in large animal models

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Gene-based treatment of motor neuron diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712590903514074

10.1517/14712590903514074

21 January 2018

Identifiers

DOI

1 reference

22 August 2017

1 reference