(Q48633842)

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

10 March 2020

title

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

1 reference

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10 March 2020

1 reference

8

1 reference

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10 March 2020

Barbara King

5

1 reference

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10 March 2020

Marten F. Snel

6

1 reference

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10 March 2020

7

John J Hopwood

1 reference

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10 March 2020

author name string

Sofia Hassiotis

1

1 reference

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10 March 2020

Helen Beard

2

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10 March 2020

Amanda Luck

3

1 reference

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10 March 2020

Paul J Trim

4

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10 March 2020

language of work or name

English

0 references

publication date

1 June 2014

1 reference

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

European Journal of Neuroscience

10 March 2020

published in

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10 March 2020

volume

39

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10 March 2020

issue

12

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10 March 2020

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

1 reference

A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome).

21 January 2018

1 reference

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

21 January 2018

1 reference

Sorting of lysosomal proteins

21 January 2018

1 reference

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

21 January 2018

1 reference

Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease

21 January 2018

1 reference

Abnormal mannose-6-phosphate receptor trafficking impairs recombinant alpha-glucosidase uptake in Pompe disease fibroblasts

21 January 2018

1 reference

Characterization of a C57BL/6 congenic mouse strain of mucopolysaccharidosis type IIIA.

21 January 2018

1 reference

Mannose 6-phosphate receptor-mediated uptake is defective in acid sphingomyelinase-deficient macrophages: implications for Niemann-Pick disease enzyme replacement therapy

21 January 2018

1 reference

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

21 January 2018

1 reference

Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I.

21 January 2018

1 reference

Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease

21 January 2018

1 reference

Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

21 January 2018

1 reference

Mannose 6-phosphate receptors: new twists in the tale

21 January 2018

1 reference

Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice

21 January 2018

1 reference

Chemically modified beta-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII.

21 January 2018

1 reference

Emerging therapies for neurodegenerative lysosomal storage disorders - from concept to reality.

21 January 2018

1 reference

Injection of recombinant human sulfamidase into the CSF via the cerebellomedullary cistern in MPS IIIA mice.

21 January 2018

1 reference

Effect of high dose, repeated intra-cerebrospinal fluid injection of sulphamidase on neuropathology in mucopolysaccharidosis type IIIA mice.

21 January 2018

1 reference

Examination of intravenous and intra-CSF protein delivery for treatment of neurological disease.

21 January 2018

1 reference

Effect of cisternal sulfamidase delivery in MPS IIIA Huntaway dogs--a proof of principle study.

21 January 2018

1 reference

Validation of a heparan sulfate-derived disaccharide as a marker of accumulation in murine mucopolysaccharidosis type IIIA.

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors

21 January 2018

1 reference

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

21 January 2018

1 reference

Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS.

21 January 2018

1 reference

Therapeutic potential of intracerebroventricular replacement of modified human β-hexosaminidase B for GM2 gangliosidosis

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Prevalence of lysosomal storage disorders

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Effects of enzyme replacement therapy on growth in patients with mucopolysaccharidosis type II.

21 January 2018

1 reference

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

21 January 2018

1 reference

Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations

21 January 2018

1 reference

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

21 January 2018

1 reference

Secondary accumulation of gangliosides in lysosomal storage disorders

21 January 2018

1 reference

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

21 January 2018

1 reference

21 January 2018

Identifiers

DOI

10.1111/EJN.12557

1 reference

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

10 March 2020

1 reference