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

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

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

http://www.wikidata.org/entity/Q34335172

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Alpha-mannosidosis A key enzyme in the biogenesis of lysosomes is a protease that regulates cholesterol metabolism Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.Cerebellar alterations and gait defects as therapeutic outcome measures for enzyme replacement therapy in α-mannosidosis.Effective intravenous therapy for neurodegenerative disease with a therapeutic enzyme and a peptide that mediates delivery to the brain Impaired lysosomal trimming of N-linked oligosaccharides leads to hyperglycosylation of native lysosomal proteins in mice with alpha-mannosidosis.Engineering of GlcNAc-1-Phosphotransferase for Production of Highly Phosphorylated Lysosomal Enzymes for Enzyme Replacement Therapy.Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.New strategies for enzyme replacement therapy for lysosomal storage diseases Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy.Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptors The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases Epinephrine enhances lysosomal enzyme delivery across the blood brain barrier by up-regulation of the mannose 6-phosphate receptor Lysosomal dysfunction causes neurodegeneration in mucolipidosis II 'knock-in' mice.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 Chemically modified beta-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII.Pharmacologic manipulation of lysosomal enzyme transport across the blood-brain barrier.Emerging therapies for neurodegenerative lysosomal storage disorders - from concept to reality.Alpha-mannosidosis: a report of 2 siblings and review of the literature.Current and emerging management options for patients with Morquio A syndrome Animal models for metabolic, neuromuscular and ophthalmological rare diseases.Proton nuclear magnetic resonance spectroscopic detection of oligomannosidic n glycans in alpha-mannosidosis: a method of monitoring treatment.High-dose enzyme replacement therapy in murine Hurler syndrome Identification of 83 novel alpha-mannosidosis-associated sequence variants: functional analysis of MAN2B1 missense mutations.Cerebral Magnetic Resonance Spectroscopy Demonstrates Long-Term Effect of Bone Marrow Transplantation in α-Mannosidosis Enzyme replacement therapy for alpha-mannosidosis: 12 months follow-up of a single centre, randomised, multiple dose study.Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy.Human α-mannosidase produced in transgenic tobacco plants is processed in human α-mannosidosis cell lines.IDS crossing of the blood-brain barrier corrects CNS defects in MPSII mice.Transport of arylsulfatase A across the blood-brain barrier in vitro.Induction of tolerance to human arylsulfatase A in a mouse model of metachromatic leukodystrophy.Swainsonine as a lysosomal toxin affects dopaminergic neurons.The effects of early and late bone marrow transplantation in siblings with alpha-mannosidosis. Is early haematopoietic cell transplantation the preferred treatment option?RTB lectin-mediated delivery of lysosomal α-l-iduronidase mitigates disease manifestations systemically including the central nervous system.Delivering drugs to the central nervous system: an overview.Alpha-Mannosidosis: Therapeutic Strategies.Idursulfase: enzyme replacement therapy for mucopolysaccharidosis Type II (Hunter syndrome)

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

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2004 nî lūn-bûn

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