Attenuation of ganglioside GM1 accumulation in the brain of GM1 gangliosidosis mice by neonatal intravenous gene transfer.
about
Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan.Evaluation of N-nonyl-deoxygalactonojirimycin as a pharmacological chaperone for human GM1 gangliosidosis leads to identification of a feline model suitable for testing enzyme enhancement therapy.Effects of cyclodextrins on GM1-gangliosides in fibroblasts from GM1-gangliosidosis patients.Gene therapy for GM1 gangliosidosis: challenges of translational medicineTransient high-level expression of beta-galactosidase after transfection of fibroblasts from GM1 gangliosidosis patients with plasmid DNA.Substrate reduction reduces gangliosides in postnatal cerebrum-brainstem and cerebellum in GM1 gangliosidosis mice.Chemical chaperone therapy: chaperone effect on mutant enzyme and cellular pathophysiology in β-galactosidase deficiency.
P2860
Q27300989-9CCE4741-5483-4D1F-97F2-A0E2F9543917Q33568024-DB39DF10-9231-41C5-94A0-780018E92E5FQ35597034-CD1197F7-08C1-4B55-9C08-5D8CB5350301Q40858975-02FD5926-25C0-4935-95DB-758946B4DAC1Q44072293-9EF15F27-1F3D-42C4-A215-CADAD34BF789Q45248629-AF787C67-7E8E-4CAE-8D28-003E9899EE01Q53251808-7578F42D-AD43-4F2D-9C01-E1521C539AA3
P2860
Attenuation of ganglioside GM1 accumulation in the brain of GM1 gangliosidosis mice by neonatal intravenous gene transfer.
description
2003 nî lūn-bûn
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2003年の論文
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2003年学术文章
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name
Attenuation of ganglioside GM1 ...... tal intravenous gene transfer.
@en
Attenuation of ganglioside GM1 ...... tal intravenous gene transfer.
@nl
type
label
Attenuation of ganglioside GM1 ...... tal intravenous gene transfer.
@en
Attenuation of ganglioside GM1 ...... tal intravenous gene transfer.
@nl
prefLabel
Attenuation of ganglioside GM1 ...... tal intravenous gene transfer.
@en
Attenuation of ganglioside GM1 ...... tal intravenous gene transfer.
@nl
P2093
P2860
P356
P1433
P1476
Attenuation of ganglioside GM1 ...... tal intravenous gene transfer.
@en
P2093
P2860
P2888
P304
P356
10.1038/SJ.GT.3302033
P577
2003-08-01T00:00:00Z