| scholarly article | Q13442814 |
| review article | Q7318358 |
| P356 | DOI | 10.1016/S0733-8619(02)00010-5 |
| P698 | PubMed publication ID | 12432834 |
| P2093 | author name string | Miguel Sena-Esteves | |
| Edward M Kaye | |||
| P2860 | cites work | Overexpression of human alpha-galactosidase A results in its intracellular aggregation, crystallization in lysosomes, and selective secretion | Q24337557 |
| The Vpr protein of human immunodeficiency virus type 1 influences nuclear localization of viral nucleic acids in nondividing host cells | Q24564509 | ||
| A simplified system for generating recombinant adenoviruses | Q24682328 | ||
| A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells | Q28249434 | ||
| In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector | Q29547503 | ||
| A third-generation lentivirus vector with a conditional packaging system | Q29616120 | ||
| Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo | Q29616612 | ||
| Engraftment of genetically engineered amniotic epithelial cells corrects lysosomal storage in multiple areas of the brain in mucopolysaccharidosis type VII mice | Q32124905 | ||
| Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. | Q33643620 | ||
| Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers | Q33782809 | ||
| Development of a self-inactivating lentivirus vector. | Q33784841 | ||
| Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo | Q33811409 | ||
| Concatamerization of adeno-associated virus circular genomes occurs through intermolecular recombination. | Q33823450 | ||
| Lentiviral vectors: turning a deadly foe into a therapeutic agent | Q33840989 | ||
| Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo | Q33844362 | ||
| Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial | Q33941289 | ||
| Enzyme replacement therapy in Fabry disease: a randomized controlled trial | Q33949356 | ||
| A novel system for the production of fully deleted adenovirus vectors that does not require helper adenovirus | Q33952908 | ||
| Perivascular microglial cells of the CNS are bone marrow-derived and present antigen in vivo | Q34049368 | ||
| Construction and applications of a highly transmissible murine retrovirus shuttle vector | Q34255941 | ||
| Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease | Q34311947 | ||
| Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors | Q34460148 | ||
| Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice | Q34483749 | ||
| Prevalence of lysosomal storage disorders | Q34489300 | ||
| Optimization of the helper-dependent adenovirus system for production and potency in vivo | Q34996749 | ||
| Phosphohexosyl components of a lysosomal enzyme are recognized by pinocytosis receptors on human fibroblasts | Q35027280 | ||
| Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. | Q35111738 | ||
| Bone marrow transplantation corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease | Q35147265 | ||
| 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 | Q35161530 | ||
| Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres | Q35254540 | ||
| Retroviral-mediated transfer of the human glucocerebrosidase gene into cultured Gaucher bone marrow | Q35604723 | ||
| Marrow stromal cells migrate throughout forebrain and cerebellum, and they differentiate into astrocytes after injection into neonatal mouse brains | Q35630632 | ||
| Anatomical evidence for cerebellar and basal ganglia involvement in higher cognitive function | Q42492293 | ||
| Expression of the lacZ reporter gene in the rat basal forebrain, hippocampus, and nigrostriatal pathway using a nonreplicating herpes simplex vector | Q42517893 | ||
| Generation of high-titer defective HSV-1 vectors using an IE 2 deletion mutant and quantitative study of expression in cultured cortical cells. | Q42815788 | ||
| Retroviral-mediated transfer of the galactocerebrosidase gene in neural progenitor cells | Q42823108 | ||
| A defective HSV-1 vector expresses Escherichia coli beta-galactosidase in cultured peripheral neurons | Q43244050 | ||
| Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model | Q43829645 | ||
| Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors | Q43867083 | ||
| Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations | Q43890879 | ||
| Oncolytic virus therapy of multiple tumors in the brain requires suppression of innate and elicited antiviral responses | Q43941683 | ||
| Lentiviral gene transfer to the nonhuman primate brain | Q44028201 | ||
| Gene transfer into experimental brain tumors mediated by adenovirus, herpes simplex virus, and retrovirus vectors | Q44221281 | ||
| Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. | Q44267005 | ||
| The herpes simplex virus amplicon: A new eucaryotic defective-virus cloning-amplifying vector | Q44397432 | ||
| Transplantation to the rat brain of human neural progenitors that were genetically modified using adenoviruses | Q44515156 | ||
| In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors. | Q45091712 | ||
| Herpesvirus vector gene transfer and expression of beta-glucuronidase in the central nervous system of MPS VII mice | Q45175987 | ||
| Direct delivery of leptin to the hypothalamus using recombinant adeno-associated virus vectors results in increased therapeutic efficacy | Q45222289 | ||
| Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII. | Q45738002 | ||
| 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 | Q45741506 | ||
| Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization | Q45742577 | ||
| A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation | Q45742580 | ||
| Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse | Q45743729 | ||
| Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector | Q45747695 | ||
| Deletion of multiple immediate-early genes from herpes simplex virus reduces cytotoxicity and permits long-term gene expression in neurons | Q45751217 | ||
| 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. | Q45751235 | ||
| 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. | Q45756120 | ||
| Prolonged in vivo gene expression driven by a tyrosine hydroxylase promoter in a defective herpes simplex virus amplicon vector | Q45766202 | ||
| Long-term expression of a foreign gene from a unique position in the latent herpes simplex virus genome | Q45767600 | ||
| 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 | Q45769549 | ||
| Gene delivery into the brain using virus vectors | Q45773320 | ||
| Delivery of virus-sized iron oxide particles to rodent CNS neurons | Q45781597 | ||
| Adenovirus-mediated gene transfer results in decreased lysosomal storage in brain and total correction in liver of aspartylglucosaminuria (AGU) mouse | Q45856025 | ||
| 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 | Q45859200 | ||
| 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. | Q45864505 | ||
| Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors | Q45867424 | ||
| 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 | Q45872484 | ||
| An adenovirus vector for gene transfer into neurons and glia in the brain | Q45874317 | ||
| Direct in vivo gene transfer to ependymal cells in the central nervous system using recombinant adenovirus vectors | Q45875094 | ||
| In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice | Q45875261 | ||
| Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors | Q45884182 | ||
| Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation | Q48110455 | ||
| A model system for in vivo gene transfer into the central nervous system using an adenoviral vector | Q48325479 | ||
| Extensive beta-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain | Q48342498 | ||
| Enzyme-replacement therapy in mucopolysaccharidosis I. | Q48705361 | ||
| Follow-up of nine patients with Hurler syndrome after bone marrow transplantation. | Q50504785 | ||
| Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors. | Q51975070 | ||
| The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher's disease. | Q52136965 | ||
| Comparative efficacy of dose regimens in enzyme replacement therapy of type I Gaucher disease. | Q52163978 | ||
| HSV/AAV hybrid amplicon vectors extend transgene expression in human glioma cells. | Q55478709 | ||
| Specific Patterns of Defective HSV-1 Gene Transfer in the Adult Central Nervous System: Implications for Gene Targeting | Q58822234 | ||
| Treatment of Late Infantile Metachromatic Leukodystrophy by Bone Marrow Transplantation | Q61769085 | ||
| Transfer of a foreign gene into the brain using adenovirus vectors | Q64377820 | ||
| Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease | Q70144770 | ||
| Long-term follow-up of the first successful bone marrow transplantation in Gaucher disease | Q70396227 | ||
| 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 | Q35757133 | ||
| 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. | Q35831286 | ||
| Utilization of the herpes simplex virus type 1 latency-associated regulatory region to drive stable reporter gene expression in the nervous system. | Q35881379 | ||
| Toxicity associated with repeated administration of first-generation adenovirus vectors does not occur with a helper-dependent vector. | Q35943949 | ||
| Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice | Q35995307 | ||
| Engraftment and migration of human bone marrow stromal cells implanted in the brains of albino rats--similarities to astrocyte grafts | Q36012180 | ||
| An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene | Q36163092 | ||
| Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells | Q36288334 | ||
| Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector | Q36586077 | ||
| Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection | Q36720691 | ||
| 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 | Q36757549 | ||
| Feline immunodeficiency virus vectors persistently transduce nondividing airway epithelia and correct the cystic fibrosis defect | Q36807130 | ||
| Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. | Q37181003 | ||
| Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector | Q37251114 | ||
| Lysosomal storage diseases | Q37285237 | ||
| 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 | Q37319132 | ||
| A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase | Q37551841 | ||
| Mannose-specific oligosaccharide recognition by mononuclear phagocytes | Q38354189 | ||
| Animal models for lysosomal storage diseases: their past and future contribution | Q38688957 | ||
| An enhanced packaging system for helper-dependent herpes simplex virus vectors | Q39580097 | ||
| Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector | Q39611260 | ||
| Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus | Q40037960 | ||
| Cytotoxicity of a replication-defective mutant of herpes simplex virus type 1 | Q40065369 | ||
| Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease | Q40600194 | ||
| Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo | Q40606944 | ||
| Inherited disorders of lysosomal metabolism | Q40690248 | ||
| Production and neurotropism of lentivirus vectors pseudotyped with lyssavirus envelope glycoproteins. | Q40788905 | ||
| Primary adult human astrocytes as an ex vivo vehicle for beta-glucuronidase delivery in the brain | Q40799301 | ||
| 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 | Q40800128 | ||
| Efficient FLPe recombinase enables scalable production of helper-dependent adenoviral vectors with negligible helper-virus contamination | Q40802046 | ||
| Development of a FLP/frt system for generating helper-dependent adenoviral vectors | Q40805792 | ||
| Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease | Q40846012 | ||
| Efficient transduction of nondividing cells by optimized feline immunodeficiency virus vectors. | Q40863212 | ||
| Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach | Q40870377 | ||
| 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 | ||
| Efficient lentiviral transduction of liver requires cell cycling in vivo | Q40909303 | ||
| A high-efficiency Cre/loxP-based system for construction of adenoviral vectors | Q40917447 | ||
| Enzymatic correction and cross-correction of mucopolysaccharidosis type I fibroblasts by adeno-associated virus-mediated transduction of the alpha-L-iduronidase gene | Q40926917 | ||
| 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 | Q40982826 | ||
| 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 | Q40984259 | ||
| 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 | Q41102578 | ||
| Expression of human beta-hexosaminidase alpha-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells | Q41212883 | ||
| Migratory patterns of lac-z transfected human glioma cells in the rat brain | Q41295339 | ||
| Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain | Q41358776 | ||
| Migration of genetically labeled glioma cells after implantation into murine brain | Q41459068 | ||
| Human immunodeficiency virus infection of cells arrested in the cell cycle | Q41531689 | ||
| Local protective effects of nerve growth factor-secreting fibroblasts against excitotoxic lesions in the rat striatum | Q41576332 | ||
| Quantitative correlation between the residual activity of beta-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease | Q41638167 | ||
| Immortalized neural progenitor cells for CNS gene transfer and repair. | Q41639955 | ||
| Long-term cross-species brain transplantation of a polymer-encapsulated dopamine-secreting cell line | Q41692307 | ||
| Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain | Q42280576 | ||
| The organization of cerebellar and basal ganglia outputs to primary motor cortex as revealed by retrograde transneuronal transport of herpes simplex virus type 1. | Q42467693 | ||
| Multiple Output Channels in the Basal Ganglia | Q42482773 | ||
| P433 | issue | 3 | |
| P921 | main subject | gene therapy | Q213901 |
| P304 | page(s) | 879-901 | |
| P577 | publication date | 2002-08-01 | |
| P1433 | published in | Neurologic Clinics | Q15756619 |
| P1476 | title | Gene therapy for the central nervous system in the lysosomal storage disorders | |
| P478 | volume | 20 |
| Q46455025 | AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease |
| Q35923476 | Gene therapy for lysosomal storage diseases (LSDs) in large animal models |
| Q45026873 | Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis. |
| Q43070160 | The Gordon Wilson lecture: using genetic medicine to regenerate diseased organs and protect against the hostile environment |
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