AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.
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Biology of adeno-associated viral vectors in the central nervous systemMolecular beacon genotyping for globoid cell leukodystrophy from hair roots in the twitcher mouse and rhesus macaqueTherapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophyTherapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIBDifferential adeno-associated virus mediated gene transfer to sensory neurons following intrathecal delivery by direct lumbar puncture.Diffusion tensor imaging detects axonal injury and demyelination in the spinal cord and cranial nerves of a murine model of globoid cell leukodystrophy.Gene therapy for lysosomal storage disorders: a good start.Effect of vitamin D3 intake on the onset of disease in a murine model of human Krabbe disease.Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis.Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophyNeural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy.CNS-directed gene therapy for lysosomal storage diseasesBone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy.Mesenchymal lineage stem cells have pronounced anti-inflammatory effects in the twitcher mouse model of Krabbe's diseaseBone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophyHigh-throughput screening of stem cell therapy for globoid cell leukodystrophy using automated neurophenotyping of twitcher mice.Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease.Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher MiceMultipotent stromal cells alleviate inflammation, neuropathology, and symptoms associated with globoid cell leukodystrophy in the twitcher mouse.Large animal models of neurological disorders for gene therapy.Hematopoietic Stem cell transplantation and lentiviral vector-based gene therapy for Krabbe's disease: Present convictions and future prospects.Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplantEffects of treatments on inflammatory and apoptotic markers in the CNS of mice with globoid cell leukodystrophy.Advances and pitfalls of cell therapy in metabolic leukodystrophies.Development of Novel Recombinant AAV Vectors and Strategies for the Potential Gene Therapy of Hemophilia.Gene therapy for the neurological manifestations in lysosomal storage disorders.AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Myelin repair by transplantation of myelin-forming cells in globoid cell leukodystrophy.Krabbe disease: One Hundred years from the bedside to the bench to the bedside.Treatment for Krabbe's disease: Finding the combination.Perspective on innovative therapies for globoid cell leukodystrophy.Immunological considerations for treating globoid cell leukodystrophy.HIV Tat Domain Improves Cross-correction of Human Galactocerebrosidase in a Gene- and Flanking Sequence-dependent Manner.Gene and stem cell therapy: alone or in combination?Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer.Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy.A HILIC-MS/MS method for simultaneous quantification of the lysosomal disease markers galactosylsphingosine and glucosylsphingosine in mouse serum
P2860
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P2860
AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.
description
2005 nî lūn-bûn
@nan
2005年の論文
@ja
2005年学术文章
@wuu
2005年学术文章
@zh-cn
2005年学术文章
@zh-hans
2005年学术文章
@zh-my
2005年学术文章
@zh-sg
2005年學術文章
@yue
2005年學術文章
@zh
2005年學術文章
@zh-hant
name
AAV2/5 vector expressing galac ...... hy more efficiently than AAV2.
@en
AAV2/5 vector expressing galac ...... hy more efficiently than AAV2.
@nl
type
label
AAV2/5 vector expressing galac ...... hy more efficiently than AAV2.
@en
AAV2/5 vector expressing galac ...... hy more efficiently than AAV2.
@nl
prefLabel
AAV2/5 vector expressing galac ...... hy more efficiently than AAV2.
@en
AAV2/5 vector expressing galac ...... hy more efficiently than AAV2.
@nl
P2093
P1433
P1476
AAV2/5 vector expressing galac ...... phy more efficiently than AAV2
@en
P2093
Carole Vogler
Corinne R Fantz
Darshong Lin
David A Wenger
Mohammad A Rafi
P304
P356
10.1016/J.YMTHE.2005.04.019
P50
P577
2005-09-01T00:00:00Z