Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII. - Wikidata - awgldk - TriplyDB

Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

http://www.wikidata.org/entity/Q34078512

about

Gene therapy for mucopolysaccharidosis Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system Clinical course of sly syndrome (mucopolysaccharidosis type VII)CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)Acid beta-glucosidase: insights from structural analysis and relevance to Gaucher disease therapy Protein replacement therapy partially corrects the cholesterol-storage phenotype in a mouse model of Niemann-Pick type C2 disease Inflammation-associated extracellular β-glucuronidase alters cellular responses to the chemical carcinogen benzo[a]pyrene Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder.Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits.Effective intravenous therapy for neurodegenerative disease with a therapeutic enzyme and a peptide that mediates delivery to the brain Human recombinant palmitoyl-protein thioesterase-1 (PPT1) for preclinical evaluation of enzyme replacement therapy for infantile neuronal ceroid lipofuscinosis.Genistein reduces glycosaminoglycan levels in a mouse model of mucopolysaccharidosis type II Physiologic and metabolic safety of butyrylcholinesterase gene therapy in mice Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood-brain barrier in the newborn mouse.A breach in the blood-brain barrier.Agile delivery of protein therapeutics to CNS New strategies for enzyme replacement therapy for lysosomal storage diseases Galactocerebrosidase-deficient oligodendrocytes maintain stable central myelin by exogenous replacement of the missing enzyme in mice Gene silencing of EXTL2 and EXTL3 as a substrate deprivation therapy for heparan sulphate storing mucopolysaccharidoses.Systemic administration of tripeptidyl peptidase I in a mouse model of late infantile neuronal ceroid lipofuscinosis: effect of glycan modification.Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy.Enzyme replacement therapy in newborn mucopolysaccharidosis IVA mice: early treatment rescues bone lesions?Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptors Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA.Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice.From the liver to the brain across the blood-brain barrier.Epinephrine enhances lysosomal enzyme delivery across the blood brain barrier by up-regulation of the mannose 6-phosphate receptor Gene therapy for lysosomal storage diseases (LSDs) in large animal models.Effect of neonatal gene therapy on lumbar spine disease in mucopolysaccharidosis VII dogs Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II Long circulating enzyme replacement therapy rescues bone pathology in mucopolysaccharidosis VII murine model.Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII.Chronic enzyme replacement therapy ameliorates neuropathology in alpha-mannosidosis mice Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome Chemically modified beta-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII.An innovative approach to the treatment of Gaucher disease and possibly other metabolic disorders of the brain.Genetic diseases of sphingolipid metabolism: pathological mechanisms and therapeutic options.

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Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

http://www.wikidata.org/entity/Q34078512

description

2005 nî lūn-bûn

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2005 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած

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2005 թվականի սեպտեմբերին հրատարակված գիտական հոդված

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2005年の論文

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2005年学术文章

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2005年学术文章

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2005年学术文章

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2005年学术文章

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2005年学术文章

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2005年學術文章

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name

Overcoming the blood-brain bar ...... ine mucopolysaccharidosis VII.

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Overcoming the blood-brain bar ...... ine mucopolysaccharidosis VII.

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Overcoming the blood-brain bar ...... ine mucopolysaccharidosis VII.

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Overcoming the blood-brain bar ...... ine mucopolysaccharidosis VII.

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Overcoming the blood-brain bar ...... ine mucopolysaccharidosis VII.

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Overcoming the blood-brain bar ...... ine mucopolysaccharidosis VII.

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Overcoming the blood-brain bar ...... ine mucopolysaccharidosis VII.

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PNAS.0506892102

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Proceedings of the National Academy of Sciences of the United States of America

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Overcoming the blood-brain bar ...... ine mucopolysaccharidosis VII.

@en

P2093

Beth Levy

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Carole Vogler

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Emil Kakkis

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Jeffrey H Grubb

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Nadine Pavloff

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Nancy Galvin

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William S Sly

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Yun Tan

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P2860

Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency

Mannose 6-phosphate receptors: new twists in the tale

Enzyme replacement therapy in a mouse model of aspartylglycosaminuria

Immune response to enzyme replacement therapy in lysosomal storage disorder patients and animal models.

Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors.

Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis.

Efficacy of enzyme replacement therapy in alpha-mannosidosis mice: a preclinical animal study.

Active site mutant transgene confers tolerance to human beta-glucuronidase without affecting the phenotype of MPS VII mice.

Murine mucopolysaccharidosis VIL: impact of therapies on the phenotype, clinical course, and pathology in a model of a lysosomal storage disease.

Mannose receptor.

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10.1073/PNAS.0506892102

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41

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102

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blood–brain barrier

Enzyme replacement therapy

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1253584

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