Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII.
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Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs.Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice.Guanidinylated neomycin mediates heparan sulfate-dependent transport of active enzymes to lysosomes.Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in α-mannosidosis.Enhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome.A breach in the blood-brain barrier.Enzyme replacement therapy for the mucopolysaccharide storage disorders.Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptorsEpinephrine enhances lysosomal enzyme delivery across the blood brain barrier by up-regulation of the mannose 6-phosphate receptorMannose receptor-mediated delivery of moss-made α-galactosidase A efficiently corrects enzyme deficiency in Fabry micePharmacologic manipulation of lysosomal enzyme transport across the blood-brain barrier.Infused Fc-tagged beta-glucuronidase crosses the placenta and produces clearance of storage in utero in mucopolysaccharidosis VII mice.Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII miceTransplantation of human umbilical mesenchymal stem cells cures the corneal defects of mucopolysaccharidosis VII mice.Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier.Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders.New biotechnological and nanomedicine strategies for treatment of lysosomal storage disordersInsulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts.Lipoprotein receptor binding, cellular uptake, and lysosomal delivery of fusions between the receptor-associated protein (RAP) and alpha-L-iduronidase or acid alpha-glucosidase.Mannose 6-phosphate receptor-mediated uptake is defective in acid sphingomyelinase-deficient macrophages: implications for Niemann-Pick disease enzyme replacement therapy.Pathogenesis and treatment of spine disease in the mucopolysaccharidoses.Recombinant encapsulated cells overexpressing alpha-L-iduronidase correct enzyme deficiency in human mucopolysaccharidosis type I cells.Numerous transcriptional alterations in liver persist after short-term enzyme-replacement therapy in a murine model of mucopolysaccharidosis type VII.Conjugation of mannose 6-phosphate-containing oligosaccharides to acid alpha-glucosidase improves the clearance of glycogen in pompe mice.A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I.
P2860
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P2860
Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII.
description
2001 nî lūn-bûn
@nan
2001年の論文
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2001年学术文章
@wuu
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
2001年學術文章
@zh-hant
name
Biodistribution, kinetics, and ...... of mucopolysaccharidosis VII.
@en
Biodistribution, kinetics, and ...... of mucopolysaccharidosis VII.
@nl
type
label
Biodistribution, kinetics, and ...... of mucopolysaccharidosis VII.
@en
Biodistribution, kinetics, and ...... of mucopolysaccharidosis VII.
@nl
prefLabel
Biodistribution, kinetics, and ...... of mucopolysaccharidosis VII.
@en
Biodistribution, kinetics, and ...... of mucopolysaccharidosis VII.
@nl
P2093
P2860
P356
P1476
Biodistribution, kinetics, and ...... l of mucopolysaccharidosis VII
@en
P2093
P2860
P304
43160-43165
P356
10.1074/JBC.M107778200
P407
P50
P577
2001-09-18T00:00:00Z