Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice.
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SUMF1 enhances sulfatase activities in vivo in five sulfatase deficienciesFemale mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field testReplacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.Characteristics of compounds that cross the blood-brain barrierMouse models of neurological disorders: a view from the blood-brain barrier.Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood-brain barrier in the newborn mouse.Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.Systemic administration of tripeptidyl peptidase I in a mouse model of late infantile neuronal ceroid lipofuscinosis: effect of glycan modification.Enzyme replacement therapy in newborn mucopolysaccharidosis IVA mice: early treatment rescues bone lesions?Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA.Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice.Chronic enzyme replacement therapy ameliorates neuropathology in alpha-mannosidosis miceMucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10Pharmacologic manipulation of lysosomal enzyme transport across the blood-brain barrier.A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA.Continual Low-Dose Infusion of Sulfamidase Is Superior to Intermittent High-Dose Delivery in Ameliorating Neuropathology in the MPS IIIA Mouse BrainPreferred transduction with AAV8 and AAV9 via thalamic administration in the MPS IIIB model: A comparison of four rAAV serotypes.Glycosaminoglycan levels and structure in a mucopolysaccharidosis IIIA mice and the effect of a highly secreted sulfamidase engineered to cross the blood-brain barrier.Embryonic stem cell-derived glial precursors as a vehicle for sulfamidase production in the MPS-IIIA mouse brain.Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice.Whole Body and CNS Biodistribution of rhHNS in Cynomolgus Monkeys after Intrathecal Lumbar Administration: Treatment Implications for Patients with MPS IIIA.Low-dose, continuous enzyme replacement therapy ameliorates brain pathology in the neurodegenerative lysosomal disorder mucopolysaccharidosis type IIIA.Inhibition of glycosaminoglycan synthesis using rhodamine B in a mouse model of mucopolysaccharidosis type IIIA.Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age.Disease stage determines the efficacy of treatment of a paediatric neurodegenerative disease.Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy.
P2860
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P2860
Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice.
description
2004 nî lūn-bûn
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2004年学术文章
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2004年学术文章
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2004年学术文章
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name
Enzyme-replacement therapy fro ...... ysaccharidosis type IIIA mice.
@en
Enzyme-replacement therapy fro ...... ysaccharidosis type IIIA mice.
@nl
type
label
Enzyme-replacement therapy fro ...... ysaccharidosis type IIIA mice.
@en
Enzyme-replacement therapy fro ...... ysaccharidosis type IIIA mice.
@nl
prefLabel
Enzyme-replacement therapy fro ...... ysaccharidosis type IIIA mice.
@en
Enzyme-replacement therapy fro ...... ysaccharidosis type IIIA mice.
@nl
P2860
P1433
P1476
Enzyme-replacement therapy fro ...... ysaccharidosis type IIIA mice.
@en
P2093
Briony L Gliddon
John J Hopwood
P2860
P356
10.1203/01.PDR.0000129661.40499.12
P407
P577
2004-05-05T00:00:00Z
P6179
1043292903