Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts.
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Gene therapy for mucopolysaccharidosisTherapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs.Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII micePhenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer.Enzyme replacement therapy for murine mucopolysaccharidosis type VIIBehavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII.From gene transfer to gene therapy in lysosomal storage diseases affecting the central nervous system.Sustained delivery of erythropoietin in mice by genetically modified skin fibroblastsActive site mutant transgene confers tolerance to human beta-glucuronidase without affecting the phenotype of MPS VII mice.Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice.Production of MPS VII mouse (Gus(tm(hE540A x mE536A)Sly)) doubly tolerant to human and mouse beta-glucuronidaseAdenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice.An approach to achieve long-term expression in skin gene therapy.Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation.Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function.Current therapies for Morquio A syndrome and their clinical outcomes.Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow.Proliferation induced by keratinocyte growth factor enhances in vivo retroviral-mediated gene transfer to mouse hepatocytes.Non-viral, integrin-mediated gene transfer into fibroblasts from patients with lysosomal storage diseases.Bilirubin glucuronidation by intact Gunn rat fibroblasts expressing bilirubin UDP-glucuronosyltransferase.Lysosomal storage diseases of animals: an essay in comparative pathology.Overexpression of Pax5 is not sufficient for neoplastic transformation of mouse neuroectoderm.Somatic gene therapy.Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery.Modification of multiple transcriptional regulatory elements in a Moloney murine leukemia virus gene transfer vector circumvents silencing in fibroblast grafts and increases levels of expression of the transferred enzyme.Single cell analysis and selection of living retrovirus vector-corrected mucopolysaccharidosis VII cells using a fluorescence-activated cell sorting-based assay for mammalian beta-glucuronidase enzymatic activity.Sustained gene expression in transplanted skin fibroblasts in rats.How realistic is cutaneous gene therapy?
P2860
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P2860
Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts.
description
1993 nî lūn-bûn
@nan
1993 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
1993 թվականի հունիսին հրատարակված գիտական հոդված
@hy
1993年の論文
@ja
1993年論文
@yue
1993年論文
@zh-hant
1993年論文
@zh-hk
1993年論文
@zh-mo
1993年論文
@zh-tw
1993年论文
@wuu
name
Correction of lysosomal storag ...... lly modified skin fibroblasts.
@ast
Correction of lysosomal storag ...... lly modified skin fibroblasts.
@en
Correction of lysosomal storag ...... lly modified skin fibroblasts.
@nl
type
label
Correction of lysosomal storag ...... lly modified skin fibroblasts.
@ast
Correction of lysosomal storag ...... lly modified skin fibroblasts.
@en
Correction of lysosomal storag ...... lly modified skin fibroblasts.
@nl
prefLabel
Correction of lysosomal storag ...... lly modified skin fibroblasts.
@ast
Correction of lysosomal storag ...... lly modified skin fibroblasts.
@en
Correction of lysosomal storag ...... lly modified skin fibroblasts.
@nl
P2093
P356
P1433
P1476
Correction of lysosomal storag ...... lly modified skin fibroblasts.
@en
P2093
P2860
P2888
P304
P356
10.1038/NG0693-154
P407
P577
1993-06-01T00:00:00Z