Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models.
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The glial response to intracerebrally delivered therapies for neurodegenerative disorders: is this a critical issue?Neuroimmune mechanisms in Krabbe's diseaseLysosomal Re-acidification Prevents Lysosphingolipid-Induced Lysosomal Impairment and Cellular ToxicityCNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorderTLR2 activation inhibits embryonic neural progenitor cell proliferationDevelopment of a New Tool for 3D Modeling for Regenerative MedicineTherapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophyAAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survivalExperimental therapies in the murine model of globoid cell leukodystrophy.Gene therapy for lysosomal storage disorders: a good start.Dynamic Activity of miR-125b and miR-93 during Murine Neural Stem Cell Differentiation In Vitro and in the Subventricular Zone Neurogenic Niche.Neural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy.Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase.Altered Trafficking and Processing of GALC Mutants Correlates with Globoid Cell Leukodystrophy SeverityRecent advances in RNA interference therapeutics for CNS diseases.Missense mutation in mouse GALC mimics human gene defect and offers new insights into Krabbe diseasePervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapyMyelin lesions associated with lysosomal and peroxisomal disorders.Genetic therapy for the nervous system.Advances and pitfalls of cell therapy in metabolic leukodystrophies.MicroRNAs and Molecular Mechanisms of Neurodegeneration.Mechanotransduction: tuning stem cells fate.Stem Cells in Neurological Disorders: Emerging Therapy with Stunning Hopes.Endothelial cell dysfunction in globoid cell leukodystrophy.Perspective on innovative therapies for globoid cell leukodystrophy.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.Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.Novel insights in basic and applied stem cell therapy.HIV Tat Domain Improves Cross-correction of Human Galactocerebrosidase in a Gene- and Flanking Sequence-dependent Manner.Gene therapy targeting oligodendrocytes provides therapeutic benefit in a leukodystrophy model.Region- and age-dependent alterations of glial-neuronal metabolic interactions correlate with CNS pathology in a mouse model of globoid cell leukodystrophy.Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy.Human iPSC-based models highlight defective glial and neuronal differentiation from neural progenitor cells in metachromatic leukodystrophy.Destabilizing Domains Enable Long-Term and Inert Regulation of GDNF Expression in the Brain
P2860
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P2860
Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models.
description
2010 nî lūn-bûn
@nan
2010年の論文
@ja
2010年学术文章
@wuu
2010年学术文章
@zh
2010年学术文章
@zh-cn
2010年学术文章
@zh-hans
2010年学术文章
@zh-my
2010年学术文章
@zh-sg
2010年學術文章
@yue
2010年學術文章
@zh-hant
name
Widespread enzymatic correctio ...... n leukodystrophy mouse models.
@en
Widespread enzymatic correctio ...... n leukodystrophy mouse models.
@nl
type
label
Widespread enzymatic correctio ...... n leukodystrophy mouse models.
@en
Widespread enzymatic correctio ...... n leukodystrophy mouse models.
@nl
prefLabel
Widespread enzymatic correctio ...... n leukodystrophy mouse models.
@en
Widespread enzymatic correctio ...... n leukodystrophy mouse models.
@nl
P2093
P50
P356
P1476
Widespread enzymatic correctio ...... n leukodystrophy mouse models.
@en
P2093
Aldo Orlacchio
Annalisa Lattanzi
Claudio Maderna
Ilaria di Girolamo
Margherita Neri
P304
P356
10.1093/HMG/DDQ099
P577
2010-03-04T00:00:00Z