AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis. (Q45884511)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:15979943%20AND%20SRC:MED&resulttype=core&format=json

13 January 2020

title

AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis (English)

1 reference

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Infantile neuronal ceroid lipofuscinosis

13 January 2020

main subject

gene therapy

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0 references

author

Shannon L Macauley

2

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13 January 2020

Judith M Ogilvie

3

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13 January 2020

Mark S Sands

4

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13 January 2020

author name string

Megan Griffey

1

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13 January 2020

publication date

1 September 2005

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13 January 2020

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13 January 2020

volume

12

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13 January 2020

issue

3

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13 January 2020

page(s)

413-421

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

13 January 2020

cites work

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Rat brain contains high levels of mannose-6-phosphorylated glycoproteins including lysosomal enzymes and palmitoyl-protein thioesterase, an enzyme implicated in infantile neuronal lipofuscinosis

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

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