Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis. (Q45026873)

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16 January 2020

title

Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis (English)

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Infantile neuronal ceroid lipofuscinosis

16 January 2020

main subject

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8

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16 January 2020

Qinwen Mao

9

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16 January 2020

author name string

Marco A Passini

1

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16 January 2020

James C Dodge

2

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16 January 2020

Jie Bu

3

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16 January 2020

Wendy Yang

4

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16 January 2020

Qi Zhao

5

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16 January 2020

Dolan Sondhi

6

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Neil R Hackett

7

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16 January 2020

Lamya S Shihabuddin

10

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Seng H Cheng

11

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David E Sleat

12

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Gregory R Stewart

13

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Beverly L Davidson

14

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Peter Lobel

15

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16 January 2020

Ronald G Crystal

16

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16 January 2020

language of work or name

English

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publication date

1 February 2006

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16 January 2020

published in

Journal of Neuroscience

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16 January 2020

volume

26

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16 January 2020

page(s)

1334-1342

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16 January 2020

issue

5

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Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice.

16 January 2020

cites work

1 reference

12 December 2020

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Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse

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Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach

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Enzyme-based diagnosis of classical late infantile neuronal ceroid lipofuscinosis: comparison of tripeptidyl peptidase I and pepstatin-insensitive protease assays

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Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors.

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Transduction of murine cerebellar neurons with recombinant FIV and AAV5 vectors

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The human CLN2 protein/tripeptidyl-peptidase I is a serine protease that autoactivates at acidic pH

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Combined injection of rAAV with mannitol enhances gene expression in the rat brain

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Binding of adeno-associated virus type 5 to 2,3-linked sialic acid is required for gene transfer

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12 December 2020

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

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12 December 2020

Production and characterization of recombinant human CLN2 protein for enzyme-replacement therapy in late infantile neuronal ceroid lipofuscinosis

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Convection-enhanced delivery of AAV-2 combined with heparin increases TK gene transfer in the rat brain.

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Targeted retrograde gene delivery for neuronal protection

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

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Gene therapy for the central nervous system in the lysosomal storage disorders

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Viral-mediated delivery of the late-infantile neuronal ceroid lipofuscinosis gene, TPP-I to the mouse central nervous system

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The neuronal ceroid-lipofuscinoses

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

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Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for canavan disease

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Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-defici

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Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model.

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12 December 2020

Basic fibroblast growth factor enhances transduction, distribution, and axonal transport of adeno-associated virus type 2 vector in rat brain

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12 December 2020

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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12 December 2020

Transduction profiles of recombinant adeno-associated virus vectors derived from serotypes 2 and 5 in the nigrostriatal system of rats

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Prevention of neuropathology in the mouse model of Hurler syndrome

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12 December 2020

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

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12 December 2020

A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration

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12 December 2020

Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.

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12 December 2020

Systemic mannitol-induced hyperosmolality amplifies rAAV2-mediated striatal transduction to a greater extent than local co-infusion

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12 December 2020

Transduction of the choroid plexus and ependyma in neonatal mouse brain by vesicular stomatitis virus glycoprotein-pseudotyped lentivirus and adeno-associated virus type 5 vectors

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

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AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy

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Restoration of aspartoacylase activity in CNS neurons does not ameliorate motor deficits and demyelination in a model of Canavan disease.

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AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease

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

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12 December 2020

AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL.

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12 December 2020

Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery.

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12 December 2020

Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease

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12 December 2020

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12 December 2020

Specific involvement of muscle, nerve, and skin in late infantile and juvenile amaurotic idiocy

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12 December 2020

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

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12 December 2020

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Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions

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12 December 2020

Selective and rapid uptake of adeno-associated virus type 2 in brain

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12 December 2020

Tripeptidyl-peptidase I is apparently the CLN2 protein absent in classical late-infantile neuronal ceroid lipofuscinosis

1 reference

12 December 2020

Classical late infantile neuronal ceroid lipofuscinosis fibroblasts are deficient in lysosomal tripeptidyl peptidase I

1 reference

12 December 2020

10.1523/JNEUROSCI.2676-05.2006

Identifiers

DOI

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