Gene therapy for the central nervous system in the lysosomal storage disorders (Q34997896)

5 August 2017

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title

Gene therapy for the central nervous system in the lysosomal storage disorders (English)

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5 August 2017

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Edward M Kaye

1

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5 August 2017

Miguel Sena-Esteves

series ordinal

2

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5 August 2017

publication date

1 August 2002

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5 August 2017

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5 August 2017

volume

20

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5 August 2017

issue

3

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5 August 2017

page(s)

879-901

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Prevalence of lysosomal storage disorders

5 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Phosphohexosyl components of a lysosomal enzyme are recognized by pinocytosis receptors on human fibroblasts

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Lysosomal storage diseases

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Quantitative correlation between the residual activity of beta-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Animal models for lysosomal storage diseases: their past and future contribution

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Treatment of Late Infantile Metachromatic Leukodystrophy by Bone Marrow Transplantation

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Overexpression of human alpha-galactosidase A results in its intracellular aggregation, crystallization in lysosomes, and selective secretion

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Inherited disorders of lysosomal metabolism

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Mannose-specific oligosaccharide recognition by mononuclear phagocytes

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Comparative efficacy of dose regimens in enzyme replacement therapy of type I Gaucher disease.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher's disease.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Enzyme-replacement therapy in mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Long-term follow-up of the first successful bone marrow transplantation in Gaucher disease

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Perivascular microglial cells of the CNS are bone marrow-derived and present antigen in vivo

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Bone marrow transplantation corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Follow-up of nine patients with Hurler syndrome after bone marrow transplantation.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Oncolytic virus therapy of multiple tumors in the brain requires suppression of innate and elicited antiviral responses

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Cytotoxicity of a replication-defective mutant of herpes simplex virus type 1

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Deletion of multiple immediate-early genes from herpes simplex virus reduces cytotoxicity and permits long-term gene expression in neurons

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Long-term expression of a foreign gene from a unique position in the latent herpes simplex virus genome

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Utilization of the herpes simplex virus type 1 latency-associated regulatory region to drive stable reporter gene expression in the nervous system.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Significantly increased expression of beta-glucuronidase in the central nervous system of mucopolysaccharidosis type VII mice from the latency-associated transcript promoter in a nonpathogenic herpes simplex virus type 1 vector

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

A defective HSV-1 vector expresses Escherichia coli beta-galactosidase in cultured peripheral neurons

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Generation of high-titer defective HSV-1 vectors using an IE 2 deletion mutant and quantitative study of expression in cultured cortical cells.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Herpes simplex virus type 1 DNA amplified as bacterial artificial chromosome in Escherichia coli: rescue of replication-competent virus progeny and packaging of amplicon vectors

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

An enhanced packaging system for helper-dependent herpes simplex virus vectors

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Preproenkephalin promoter yields region-specific and long-term expression in adult brain after direct in vivo gene transfer via a defective herpes simplex viral vector

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

A herpes simplex virus-1 vector containing the rat tyrosine hydroxylase promoter directs cell type-specific expression of beta-galactosidase in cultured rat peripheral neurons

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

HSV/AAV hybrid amplicon vectors extend transgene expression in human glioma cells.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Expression of the lacZ reporter gene in the rat basal forebrain, hippocampus, and nigrostriatal pathway using a nonreplicating herpes simplex vector

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Specific Patterns of Defective HSV-1 Gene Transfer in the Adult Central Nervous System: Implications for Gene Targeting

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Multiple Output Channels in the Basal Ganglia

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

The organization of cerebellar and basal ganglia outputs to primary motor cortex as revealed by retrograde transneuronal transport of herpes simplex virus type 1.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Anatomical evidence for cerebellar and basal ganglia involvement in higher cognitive function

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

A model system for in vivo gene transfer into the central nervous system using an adenoviral vector

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

An adenovirus vector for gene transfer into neurons and glia in the brain

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

A simplified system for generating recombinant adenoviruses

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Toxicity associated with repeated administration of first-generation adenovirus vectors does not occur with a helper-dependent vector.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

A novel system for the production of fully deleted adenovirus vectors that does not require helper adenovirus

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Development of a FLP/frt system for generating helper-dependent adenoviral vectors

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

A high-efficiency Cre/loxP-based system for construction of adenoviral vectors

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Optimization of the helper-dependent adenovirus system for production and potency in vivo

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Efficient FLPe recombinase enables scalable production of helper-dependent adenoviral vectors with negligible helper-virus contamination

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

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.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Direct delivery of leptin to the hypothalamus using recombinant adeno-associated virus vectors results in increased therapeutic efficacy

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Characterization of intrastriatal recombinant adeno-associated virus-mediated gene transfer of human tyrosine hydroxylase and human GTP-cyclohydrolase I in a rat model of Parkinson's disease.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Concatamerization of adeno-associated virus circular genomes occurs through intermolecular recombination.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Construction and applications of a highly transmissible murine retrovirus shuttle vector

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Immortalized neural progenitor cells for CNS gene transfer and repair.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Migratory patterns of lac-z transfected human glioma cells in the rat brain

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Migration of genetically labeled glioma cells after implantation into murine brain

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Human immunodeficiency virus infection of cells arrested in the cell cycle

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

The Vpr protein of human immunodeficiency virus type 1 influences nuclear localization of viral nucleic acids in nondividing host cells

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Efficient lentiviral transduction of liver requires cell cycling in vivo

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Lentiviral gene transfer to the nonhuman primate brain

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Development of a self-inactivating lentivirus vector.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Efficient transduction of nondividing cells by optimized feline immunodeficiency virus vectors.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Feline immunodeficiency virus vectors persistently transduce nondividing airway epithelia and correct the cystic fibrosis defect

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Production and neurotropism of lentivirus vectors pseudotyped with lyssavirus envelope glycoproteins.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Retroviral-mediated transfer of the human glucocerebrosidase gene into cultured Gaucher bone marrow

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Herpesvirus vector gene transfer and expression of beta-glucuronidase in the central nervous system of MPS VII mice

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Gene therapy for canine alpha-L-iduronidase deficiency: in utero adoptive transfer of genetically corrected hematopoietic progenitors results in engraftment but not amelioration of disease

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Correction of murine galactosialidosis by bone marrow-derived macrophages overexpressing human protective protein/cathepsin A under control of the colony-stimulating factor-1 receptor promoter

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Bone marrow stem cell gene therapy of arylsulfatase A-deficient mice, using an arylsulfatase A mutant that is hypersecreted from retrovirally transduced donor-type cells

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations

1 reference

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7 January 2021

Gene transfer into experimental brain tumors mediated by adenovirus, herpes simplex virus, and retrovirus vectors

1 reference

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7 January 2021

Gene delivery into the brain using virus vectors

1 reference

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7 January 2021

Delivery of virus-sized iron oxide particles to rodent CNS neurons

1 reference

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7 January 2021

Direct in vivo gene transfer to ependymal cells in the central nervous system using recombinant adenovirus vectors

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Transfer of a foreign gene into the brain using adenovirus vectors

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Extensive beta-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Adenovirus-mediated gene transfer results in decreased lysosomal storage in brain and total correction in liver of aspartylglucosaminuria (AGU) mouse

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Enzymatic correction and cross-correction of mucopolysaccharidosis type I fibroblasts by adeno-associated virus-mediated transduction of the alpha-L-iduronidase gene

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Lentiviral vectors: turning a deadly foe into a therapeutic agent

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer.

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Local protective effects of nerve growth factor-secreting fibroblasts against excitotoxic lesions in the rat striatum

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of β-glucuronidase

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Transplantation to the rat brain of human neural progenitors that were genetically modified using adenoviruses

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Expression of human beta-hexosaminidase alpha-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Retroviral-mediated transfer of the galactocerebrosidase gene in neural progenitor cells

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Engraftment and migration of human bone marrow stromal cells implanted in the brains of albino rats--similarities to astrocyte grafts

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Marrow stromal cells migrate throughout forebrain and cerebellum, and they differentiate into astrocytes after injection into neonatal mouse brains

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Primary adult human astrocytes as an ex vivo vehicle for beta-glucuronidase delivery in the brain

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Engraftment of genetically engineered amniotic epithelial cells corrects lysosomal storage in multiple areas of the brain in mucopolysaccharidosis type VII mice

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

Long-term cross-species brain transplantation of a polymer-encapsulated dopamine-secreting cell line

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

A third-generation lentivirus vector with a conditional packaging system

1 reference

https://api.crossref.org/works/10.1016/S0733-8619(02)50010-5

7 January 2021

10.1016/S0733-8619(02)00010-5

Identifiers

DOI

1 reference

5 August 2017

1 reference