Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer.
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Gene therapy for mucopolysaccharidosisTherapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs.Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage diseaseThe effect of neonatal gene therapy on skeletal manifestations in mucopolysaccharidosis VII dogs after a decade.Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidosesTherapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIBAAV-mediated liver-directed gene therapy.VEGF increases engraftment of bone marrow-derived endothelial progenitor cells (EPCs) into vasculature of newborn murine recipients.Increasing cholesterol synthesis in 7-dehydrosterol reductase (DHCR7) deficient mouse models through gene transferTransgene produces massive overexpression of human beta -glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors.Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?Intravenous injections in neonatal mice.Gene therapy progress and prospects: gene therapy of lysosomal storage disorders.Safety of adeno-associated virus gene therapy vectors: a current evaluation.β2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease.Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector.Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver.Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?Frequency and spectrum of genomic integration of recombinant adeno-associated virus serotype 8 vector in neonatal mouse liverPotential of AAV vectors in the treatment of metabolic disease.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Pathogenesis and treatment of spine disease in the mucopolysaccharidoses.Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models.Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice.Numerous transcriptional alterations in liver persist after short-term enzyme-replacement therapy in a murine model of mucopolysaccharidosis type VII.Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII.Attenuation of ganglioside GM1 accumulation in the brain of GM1 gangliosidosis mice by neonatal intravenous gene transfer.Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL.
P2860
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P2860
Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer.
description
2001 nî lūn-bûn
@nan
2001年の論文
@ja
2001年学术文章
@wuu
2001年学术文章
@zh
2001年学术文章
@zh-cn
2001年学术文章
@zh-hans
2001年学术文章
@zh-my
2001年学术文章
@zh-sg
2001年學術文章
@yue
2001年學術文章
@zh-hant
name
Prevention of systemic clinica ...... diated neonatal gene transfer.
@en
Prevention of systemic clinica ...... diated neonatal gene transfer.
@nl
type
label
Prevention of systemic clinica ...... diated neonatal gene transfer.
@en
Prevention of systemic clinica ...... diated neonatal gene transfer.
@nl
prefLabel
Prevention of systemic clinica ...... diated neonatal gene transfer.
@en
Prevention of systemic clinica ...... diated neonatal gene transfer.
@nl
P2093
P2860
P356
P1433
P1476
Prevention of systemic clinica ...... diated neonatal gene transfer.
@en
P2093
P2860
P2888
P304
P356
10.1038/SJ.GT.3301420
P577
2001-09-01T00:00:00Z
P5875
P6179
1040230855