Disease stage determines the efficacy of treatment of a paediatric neurodegenerative disease. (Q48633842)

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10 March 2020

title

Disease stage determines the efficacy of treatment of a paediatric neurodegenerative disease (English)

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10 March 2020

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8

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10 March 2020

Barbara King

5

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10 March 2020

Marten F. Snel

6

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7

John J Hopwood

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author name string

Sofia Hassiotis

1

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10 March 2020

Helen Beard

2

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Amanda Luck

3

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Paul J Trim

4

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10 March 2020

language of work or name

English

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publication date

1 June 2014

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European Journal of Neuroscience

10 March 2020

published in

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volume

39

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issue

12

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page(s)

2139-2150

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Neuroinflammatory and oxidative stress phenomena in MPS IIIA mouse model: the positive effect of long-term aspirin treatment

10 March 2020

cites work

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AAV1 mediated co-expression of formylglycine-generating enzyme and arylsulfatase a efficiently corrects sulfatide storage in a mouse model of metachromatic leukodystrophy

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Allogeneic stem cell transplantation does not improve neurological deficits in mucopolysaccharidosis type IIIA mice

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Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age.

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Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders

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Prevalence of lysosomal storage disorders

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Systemic administration of tripeptidyl peptidase I in a mouse model of late infantile neuronal ceroid lipofuscinosis: effect of glycan modification

21 January 2018

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Impact of high-dose, chemically modified sulfamidase on pathology in a murine model of MPS IIIA.

21 January 2018

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Intracerebral injection of sulfamidase delays neuropathology in murine MPS-IIIA.

21 January 2018

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Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer

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Effective gene therapy for an inherited CNS disease in a large animal model

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Secondary accumulation of gangliosides in lysosomal storage disorders

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Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB

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21 January 2018

Identifiers

DOI

10.1111/EJN.12557

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25068161%20AND%20SRC:MED&resulttype=core&format=json

10 March 2020

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