AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival (Q33728295)

28 July 2017

AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival (English)

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28 July 2017

7

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28 July 2017

Rena C Baek

1

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28 July 2017

Marike L D Broekman

2

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28 July 2017

Stanley G Leroy

3

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28 July 2017

Laryssa A Tierney

4

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28 July 2017

Michael A Sandberg

5

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28 July 2017

Alessandra d'Azzo

6

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28 July 2017

Miguel Sena-Esteves

8

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28 July 2017

language of work or name

English

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publication date

18 October 2010

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28 July 2017

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28 July 2017

5

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28 July 2017

10

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28 July 2017

e13468

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Creative Commons Attribution 4.0 International

28 July 2017

start time

18 October 2010

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April 2022 Public Data File from Crossref

copyright status

copyrighted

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ImageQuant

2 references

11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC2956705/fullTextXML

inferred from PubMed Central ID database lookup

CZ Software Mentions: A large dataset of software mentions in the biomedical literature

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Long-term AAV vector gene and protein expression in mouse brain from a small pan-cellular promoter is similar to neural cell promoters

21 June 2018

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Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity

21 June 2018

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AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method

21 June 2018

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Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse

21 June 2018

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

21 June 2018

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Effective gene therapy in an authentic model of Tay-Sachs-related diseases

21 June 2018

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21 June 2018

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Gene therapy for lysosomal storage diseases.

21 June 2018

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Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain

21 June 2018

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

21 June 2018

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Chemokine-induced recruitment of genetically modified bone marrow cells into the CNS of GM1-gangliosidosis mice corrects neuronal pathology

21 June 2018

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A simple method for the isolation and purification of total lipides from animal tissues

21 June 2018

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Beta-galactosidase-deficient mouse as an animal model for GM1-gangliosidosis

21 June 2018

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Genetic analysis of cerebellar lipids in mice susceptible to audiogenic seizures.

21 June 2018

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Myelination in rat brain: changes in myelin composition during brain maturation

21 June 2018

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Biochemical changes in mouse brain composition during myelination.

21 June 2018

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Pathologic findings in fetal GM1 gangliosidosis.

21 June 2018

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Abnormalities of cerebral lipids in GM1-gangliosidoses, infantile, juvenile, and chronic type.

21 June 2018

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Dysmyelinogenesis in animal model of GM1 gangliosidosis

21 June 2018

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Prenatal diagnosis of GM1-gangliosidosis: Biochemical manifestations in fetal tissues

21 June 2018

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Generalized gangliosidosis: beta-galactosidase deficiency.

21 June 2018

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Inheritance of lysosomal acid beta-galactosidase activity and gangliosides in crosses of DBA/2J and knockout mice.

21 January 2018

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Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis

21 January 2018

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Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis

21 January 2018

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

21 January 2018

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Complete correction of enzymatic deficiency and neurochemistry in the GM1-gangliosidosis mouse brain by neonatal adeno-associated virus-mediated gene delivery

21 January 2018

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

21 January 2018

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Mechanisms of distribution of mouse beta-galactosidase in the adult GM1-gangliosidosis brain

21 January 2018

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A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease

21 January 2018

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Factor IX variants improve gene therapy efficacy for hemophilia B.

21 January 2018

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Influence of caloric restriction on motor behavior, longevity, and brain lipid composition in Sandhoff disease mice

21 January 2018

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N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice

21 January 2018

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https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0013468

GD3 ganglioside is a glycolipid characteristic of immature neuroectodermal cells.

21 January 2018

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

21 January 2018

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Alterations in metabolism and gene expression in brain regions during cuprizone-induced demyelination and remyelination

21 January 2018

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Heterosis for brain myelin content in mice.

21 January 2018

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0013468

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

21 January 2018

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Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models

21 January 2018

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Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis

21 January 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2956705

Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2956705

Substrate reduction reduces gangliosides in postnatal cerebrum-brainstem and cerebellum in GM1 gangliosidosis mice.

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2956705

GENERALIZED GANGLIOSIDOSIS: ANOTHER INBORN ERROR OF GANGLIOSIDE METABOLISM?

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2956705

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

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2956705

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

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2956705

Infantile G(M1) gangliosidosis: complete morphology and histochemistry of two autopsy cases, with particular reference to delayed central nervous system myelination

30 October 2018

1 reference

12 December 2020

Neurochemical, morphological, and neurophysiological abnormalities in retinas of Sandhoff and GM1 gangliosidosis mice

1 reference

12 December 2020

Generalized CNS disease and massive GM1-ganglioside accumulation in mice defective in lysosomal acid beta-galactosidase

1 reference

12 December 2020

Quantitative estimation of sialic acids. II. A colorimetric resorcinol-hydrochloric acid method

1 reference

12 December 2020

Retinal Gangliosides in RCS Mutant Rats

1 reference

12 December 2020

The use of Sep-Pak C18 cartridges during the isolation of gangliosides

1 reference

12 December 2020

GM1-ganglioside-mediated activation of the unfolded protein response causes neuronal death in a neurodegenerative gangliosidosis

1 reference

12 December 2020

N-butyldeoxygalactonojirimycin reduces neonatal brain ganglioside content in a mouse model of GM1 gangliosidosis

1 reference

12 December 2020

10.1371/JOURNAL.PONE.0013468

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DOI

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28 July 2017

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28 July 2017

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