Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid - Wikidata - wikimedia - TriplyDB

Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid

Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid

http://www.wikidata.org/entity/Q30497736

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Transcranial pulsed ultrasound facilitates brain uptake of laronidase in enzyme replacement therapy for Mucopolysaccharidosis type I disease Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.Safety Evaluation of CNS Administered Biologics-Study Design, Data Interpretation, and Translation to the Clinic.AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival Human recombinant palmitoyl-protein thioesterase-1 (PPT1) for preclinical evaluation of enzyme replacement therapy for infantile neuronal ceroid lipofuscinosis.Mucopolysaccharidosis VI.Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I.Controlling brain tumor growth by intraventricular administration of an AAV vector encoding IFN-beta.Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I.Therapeutic approaches for lysosomal storage diseases Systemic administration of tripeptidyl peptidase I in a mouse model of late infantile neuronal ceroid lipofuscinosis: effect of glycan modification.Substrate deprivation therapy: a new hope for patients suffering from neuronopathic forms of inherited lysosomal storage diseases.Arterial pathology in canine mucopolysaccharidosis-I and response to therapy Candidate molecules for chemical chaperone therapy of GM1-gangliosidosis.Biomarkers in the diagnosis of lysosomal storage disorders: proteins, lipids, and inhibodies.Cognitive, medical, and neuroimaging characteristics of attenuated mucopolysaccharidosis type II.Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase.Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis.Gene therapy for neurologic manifestations of mucopolysaccharidoses.Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I.Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment.Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I.The effects of intracisternal enzyme replacement versus sham treatment on central neuropathology in preclinical canine fucosidosis Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis I.A numerical investigation of intrathecal isobaric drug dispersion within the cervical subarachnoid space Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses.Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.Glycan-based biomarkers for mucopolysaccharidoses.Automated segmentation of the canine corpus callosum for the measurement of diffusion tensor imaging The choroid plexus and cerebrospinal fluid: emerging roles in development, disease, and therapy.Review of the use of idursulfase in the treatment of mucopolysaccharidosis II.Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients.Intrathecal delivery of fluorescent labeled butyrylcholinesterase to the brains of butyrylcholinesterase knock-out mice: visualization and quantification of enzyme distribution in the brain.Genistin-rich soy isoflavone extract in substrate reduction therapy for Sanfilippo syndrome: An open-label, pilot study in 10 pediatric patients.Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts.Genetic therapy for the nervous system.Emerging therapies for neurodegenerative lysosomal storage disorders - from concept to reality.Physiology of the intrathecal bolus: the leptomeningeal route for macromolecule and particle delivery to CNS.

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Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid

http://www.wikidata.org/entity/Q30497736

description

2007 nî lūn-bûn

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2007 թուականի Փետրուարին հրատարակուած գիտական յօդուած

@hyw

2007 թվականի փետրվարին հրատարակված գիտական հոդված

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2007年の論文

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2007年論文

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2007年論文

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2007年論文

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2007年論文

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2007年論文

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2007年论文

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name

Intrathecal enzyme replacement ...... se via the cerebrospinal fluid

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Intrathecal enzyme replacement ...... se via the cerebrospinal fluid

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Intrathecal enzyme replacement ...... se via the cerebrospinal fluid

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Intrathecal enzyme replacement ...... se via the cerebrospinal fluid

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J.YMGME.2006.12.012

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Molecular Genetics and Metabolism

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Intrathecal enzyme replacement ...... se via the cerebrospinal fluid

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Beth Levy

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Carole Vogler

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Emil Kakkis

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Maryn Peinovich

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Merry Passage

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Michael McEntee

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P2860

Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease

Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease.

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome.

Convection-enhanced delivery of macromolecules in the brain.

Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis I

Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease.

Outwitting the blood-brain barrier for therapeutic purposes: osmotic opening and other means.

Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells.

Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group.

Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

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10.1016/J.YMGME.2006.12.012

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1

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cerebrospinal fluid

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3009387

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