In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice.
about
Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transferIntramuscular viral delivery of paraplegin rescues peripheral axonopathy in a model of hereditary spastic paraplegiaSUMF1 enhances sulfatase activities in vivo in five sulfatase deficienciesRetroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectivesAxonal degeneration in paraplegin-deficient mice is associated with abnormal mitochondria and impairment of axonal transportCNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorderIntrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluidGene therapy: prospects for glycolipid storage diseases.Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.Delivery of the Cre recombinase by a self-deleting lentiviral vector: efficient gene targeting in vivo.Nonneurotropic adenovirus: a vector for gene transfer to the brain and gene therapy of neurological disordersRecent advances in the pharmacology of neurological gene therapy.Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors.Advanced modular self-inactivating lentiviral expression vectors for multigene interventions in mammalian cells and in vivo transduction.A lentiviral gene therapy strategy for the in vitro production of feline erythropoietin.Gene therapy of chronic granulomatous disease: the engraftment dilemmaGene therapy for lysosomal storage disorders: a good start.Gene therapy for neurological disease.Gene therapy progress and prospects: gene therapy of lysosomal storage disorders.Metachromatic leukodystrophy: recent research developments.Neural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy.Clinical applications involving CNS gene transfer.Methods for gene transfer to the central nervous system.CNS-directed gene therapy for lysosomal storage diseasesCurrent and emerging therapies for the lysosomal storage disorders.Antiprion prophylaxis by gene transfer of a soluble prion antagonist.Arylsulfatase A Overexpressing Human iPSC-derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy.Novel candidate disease for gene therapy: metachromatic leukodystrophy.Robust in vivo gene transfer into adult mammalian neural stem cells by lentiviral vectors.Genetic therapy for the nervous system.Intraneural GJB1 gene delivery improves nerve pathology in a model of X-linked Charcot-Marie-Tooth disease.Gene delivery targeted to oligodendrocytes using a lentiviral vector.Mammalian arylsulfatase A functions as a novel component of the extracellular matrix.Loss of parkin or PINK1 function increases Drp1-dependent mitochondrial fragmentationHigh-titer lentiviral vectors stimulate fetal calf serum-specific human CD4 T-cell responses: implications in human gene therapy.Metachromatic leukodystrophy: genetics, pathogenesis and therapeutic options.Stability of lentiviral vector-mediated transgene expression in the brain in the presence of systemic antivector immune responses.Optimized lentiviral vector production and purification procedure prevents immune response after transduction of mouse brain.Stereotactic injection of microRNA-expressing lentiviruses to the mouse hippocampus ca1 region and assessment of the behavioral outcomeMitochondrial imaging in dorsal root ganglion neurons following the application of inducible adenoviral vector expressing two fluorescent proteins.
P2860
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P2860
In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice.
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
In vivo gene therapy of metach ...... impairments in affected mice.
@en
In vivo gene therapy of metach ...... impairments in affected mice.
@nl
type
label
In vivo gene therapy of metach ...... impairments in affected mice.
@en
In vivo gene therapy of metach ...... impairments in affected mice.
@nl
prefLabel
In vivo gene therapy of metach ...... impairments in affected mice.
@en
In vivo gene therapy of metach ...... impairments in affected mice.
@nl
P2093
P2860
P50
P356
P1433
P1476
In vivo gene therapy of metach ...... impairments in affected mice.
@en
P2093
A Consiglio
A Oliverio
G Benaglia
J C Bensadoun
S Marchesini
P2860
P2888
P304
P356
10.1038/85454
P407
P577
2001-03-01T00:00:00Z
P5875
P6179
1049144453