Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts? - Wikidata - wikimedia - TriplyDB

Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?

Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?

http://www.wikidata.org/entity/Q34982831

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Exome-sequencing confirms DNAJC5 mutations as cause of adult neuronal ceroid-lipofuscinosis New therapeutic approaches for Krabbe disease: The potential of pharmacological chaperones The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse.Neuroinflammatory paradigms in lysosomal storage diseases.Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy Anti-inflammatory Therapy With Simvastatin Improves Neuroinflammation and CNS Function in a Mouse Model of Metachromatic Leukodystrophy The sphingolipid psychosine inhibits fast axonal transport in Krabbe disease by activation of GSK3β and deregulation of molecular motors.Multipotent stromal cells alleviate inflammation, neuropathology, and symptoms associated with globoid cell leukodystrophy in the twitcher mouse.Farber disease: understanding a fatal childhood disorder and dissecting ceramide biology.Chaperone-mediated gene therapy with recombinant AAV-PPCA in a new mouse model of type I sialidosis Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.Factors and processes modulating phenotypes in neuronopathic lysosomal storage diseases.Platelets are efficient and protective depots for storage, distribution, and delivery of lysosomal enzyme in mice with Hurler syndrome.Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders.Considerations for the treatment of infantile neuronal ceroid lipofuscinosis (infantile Batten disease)Glucocerebrosidase inhibitors for the treatment of Gaucher disease.Gene therapy for the neurological manifestations in lysosomal storage disorders.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Nanotechnology applied to treatment of mucopolysaccharidoses.Molecular Mechanisms of Disease Pathogenesis Differ in Krabbe Disease Variants.Perspective on innovative therapies for globoid cell leukodystrophy.Oxidative stress as a therapeutic target in globoid cell leukodystrophy.A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells.Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice.Astrocytosis in infantile neuronal ceroid lipofuscinosis: friend or foe?Signatures of Altered Gene Expression in Dorsal Root Ganglia of a Fabry Disease Mouse Model.

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Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?

http://www.wikidata.org/entity/Q34982831

description

2011 nî lūn-bûn

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2011 թուականի Մարտին հրատարակուած գիտական յօդուած

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2011 թվականի մարտին հրատարակված գիտական հոդված

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2011年の論文

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2011年論文

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Combination therapies for lyso ...... ter than the sum of its parts?

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Combination therapies for lyso ...... ter than the sum of its parts?

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Combination therapies for lyso ...... ter than the sum of its parts?

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Combination therapies for lyso ...... ter than the sum of its parts?

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Combination therapies for lyso ...... ter than the sum of its parts?

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Combination therapies for lyso ...... ter than the sum of its parts?

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Combination therapies for lyso ...... ter than the sum of its parts?

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Human Molecular Genetics

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Combination therapies for lyso ...... ter than the sum of its parts?

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Adarsh S Reddy

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Jacqueline A Hawkins-Salsbury

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Mark S Sands

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P2860

Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer

NSAIDs increase survival in the Sandhoff disease mouse: synergy with N-butyldeoxynojirimycin

Inhibition of calcium uptake via the sarco/endoplasmic reticulum Ca2+-ATPase in a mouse model of Sandhoff disease and prevention by treatment with N-butyldeoxynojirimycin

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs.

Adeno-associated virus serotypes: vector toolkit for human gene therapy.

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and S

Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome.

Incidence of inborn errors of metabolism in British Columbia, 1969-1996.

Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB

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10.1093/HMG/DDR112

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