(Q45889316)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:16364693%20AND%20SRC:MED&resulttype=core&format=json

10 January 2020

title

CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis (English)

1 reference

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Infantile neuronal ceroid lipofuscinosis

10 January 2020

main subject

0 references

0 references

1 reference

8

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10 January 2020

Annie E Wentz

7

1 reference

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10 January 2020

Mark S Sands

9

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10 January 2020

author name string

Megan A Griffey

1

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10 January 2020

David Wozniak

2

1 reference

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10 January 2020

Michael Wong

3

1 reference

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10 January 2020

Ellen Bible

4

1 reference

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10 January 2020

Kendra Johnson

5

1 reference

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10 January 2020

Steven M Rothman

6

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10 January 2020

publication date

20 December 2005

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10 January 2020

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10 January 2020

volume

13

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10 January 2020

issue

3

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10 January 2020

page(s)

538-547

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The neuronal ceroid-lipofuscinoses (Batten disease): a new class of lysosomal storage diseases

10 January 2020

cites work

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Batten's disease: clues to neuronal protein catabolism in lysosomes

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Mutations in the palmitoyl protein thioesterase gene causing infantile neuronal ceroid lipofuscinosis

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Association of mutations in a lysosomal protein with classical late-infantile neuronal ceroid lipofuscinosis

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Isolation of a novel gene underlying batten disease, CLN3

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Infantile type of so-called neuronal ceroid-lipofuscinosis. 1. A clinical study of 15 patients

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Variability in the clinical and pathological findings in the neuronal ceroid lipofuscinoses: Review of data and observations

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Genetics of the neuronal ceroid lipofuscinoses

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Infantile type of so-called neuronal ceroid-lipofuscinosis. Histological and electron microscopic studies

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Infantile type of so-called neuronal ceroid-lipofuscinosis. 2. Morphological and biochemical studies

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Disruption of PPT1 or PPT2 causes neuronal ceroid lipofuscinosis in knockout mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Regional and cellular neuropathology in the palmitoyl protein thioesterase-1 null mutant mouse model of infantile neuronal ceroid lipofuscinosis

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Inborn errors of mucopolysaccharide metabolism

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Structure and function of the mannose 6-phosphate/insulinlike growth factor II receptors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Extensive beta-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Expression of immune-related molecules is downregulated in twitcher mice following bone marrow transplantation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Deletion of macrophage-inflammatory protein 1 alpha retards neurodegeneration in Sandhoff disease mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Efficient selection for high-expression transfectants with a novel eukaryotic vector

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Novel tools for production and purification of recombinant adenoassociated virus vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

A rapid fluorogenic palmitoyl-protein thioesterase assay: pre- and postnatal diagnosis of INCL

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

An improved cupric-silver method for impregnation of axonal and terminal degeneration.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Application of silver degeneration stains for neurotoxicity testing

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Impaired synaptic plasticity and cAMP response element-binding protein activation in Ca2+/calmodulin-dependent protein kinase type IV/Gr-deficient mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Pole test is a useful method for evaluating the mouse movement disorder caused by striatal dopamine depletion

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Ataxia and paroxysmal dyskinesia in mice lacking axonally transported FGF14.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Acute behavioral effects of MK-801 in the mouse.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008

Altered stress-induced anxiety in adenylyl cyclase type VIII-deficient mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.11.008