Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene
about
Engineering mesenchymal stem cells for regenerative medicine and drug deliveryPhenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer.Active site mutant transgene confers tolerance to human beta-glucuronidase without affecting the phenotype of MPS VII mice.Transgene produces massive overexpression of human beta -glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors.Treatment of inherited neurometabolic diseases: the future.A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VIIMesenchymal stem cells for the sustained in vivo delivery of bioactive factors.Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function.Biopharmaceutical drug targeting to the brain.Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector.Effect of Culture Conditions on Reference Genes Expression in Placenta-derived Stem Cells.Magnetic resonance imaging detects differences in migration between primary and immortalized neural stem cells.Exploring pathogenetic mechanisms using transgenic animals.The twitcher mouse: a model for Krabbe disease and for experimental therapies.Lysosomal storage diseases of animals: an essay in comparative pathology.Viable mouse models of acid beta-glucosidase deficiency: the defect in Gaucher diseaseLong-term AAV vector gene and protein expression in mouse brain from a small pan-cellular promoter is similar to neural cell promoters.Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer.
P2860
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P2860
Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene
description
1990 nî lūn-bûn
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1990 թուականի Մայիսին հրատարակուած գիտական յօդուած
@hyw
1990 թվականի մայիսին հրատարակված գիտական հոդված
@hy
1990年の論文
@ja
1990年論文
@yue
1990年論文
@zh-hant
1990年論文
@zh-hk
1990年論文
@zh-mo
1990年論文
@zh-tw
1990年论文
@wuu
name
Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene
@ast
Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene
@en
type
label
Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene
@ast
Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene
@en
prefLabel
Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene
@ast
Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene
@en
P2093
P2860
P356
P1476
Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene
@en
P2093
E H Birkenmeier
J W Hoffmann
P2860
P304
P356
10.1073/PNAS.87.10.3914
P407
P577
1990-05-01T00:00:00Z