(Q45409241)

English

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

scientific article published in January 2007

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scholarly article

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

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

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

0 references

Thomas N. Seyfried

5

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

author name string

M L D Broekman

1

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

R C Baek

2

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

L A Comer

3

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

J L Fernandez

4

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

M Sena-Esteves

6

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

publication date

1 January 2007

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

Molecular Therapy

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

15

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

1

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

30-37

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

Generalized gangliosidosis: beta-galactosidase deficiency.

1 reference

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Cerebral GM1-gangliosidosis: chemical pathology of visceral organs.

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

1 reference

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Pathologic findings in fetal GM1 gangliosidosis.

1 reference

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

1 reference

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

1 reference

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

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

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Neurological manifestations of knockout mice with beta-galactosidase deficiency

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Inborn errors of mucopolysaccharide metabolism

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Gene therapy for lysosomal storage diseases.

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Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase

1 reference

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

1 reference

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

1 reference

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Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector

1 reference

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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

1 reference

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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

1 reference

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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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Cholesterol modulates membrane traffic along the endocytic pathway in sphingolipid-storage diseases

1 reference

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Sphingolipid storage induces accumulation of intracellular cholesterol by stimulating SREBP-1 cleavage

1 reference

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7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300004

7 January 2021

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Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300004

7 January 2021

based on heuristic

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Attenuation of ganglioside GM1 accumulation in the brain of GM1 gangliosidosis mice by neonatal intravenous gene transfer

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300004

7 January 2021

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

1 reference

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7 January 2021

based on heuristic

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Transgene produces massive overexpression of human beta -glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors

1 reference

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7 January 2021

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Neurons in Niemann-Pick disease type C accumulate gangliosides as well as unesterified cholesterol and undergo dendritic and axonal alterations

1 reference

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7 January 2021

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Accumulation of glycosphingolipids in Niemann-Pick C disease disrupts endosomal transport

1 reference

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7 January 2021

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Outsourcing in the brain: do neurons depend on cholesterol delivery by astrocytes?

1 reference

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

1 reference

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Biochemical changes in mouse brain composition during myelination

1 reference

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

1 reference

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Dysmyelinogenesis in animal model of GM1 gangliosidosis

1 reference

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

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Magnetic resonance imaging findings and novel mutations in GM1 gangliosidosis.

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

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Molecular cloning of mouse acid beta-galactosidase cDNA: sequence, expression of catalytic activity and comparison with the human enzyme

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

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Optimized large-scale production of high titer lentivirus vector pseudotypes

1 reference

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Correction of acid beta-galactosidase deficiency in GM1 gangliosidosis human fibroblasts by retrovirus vector-mediated gene transfer: higher efficiency of release and cross-correction by the murine enzyme

1 reference

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Differential Cellular Enrichment of Gangliosides in the Mouse Cerebellum: Analysis Using Neurological Mutants

1 reference

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CHROMATOGRAPHlC SEPARATION OF HUMAN BRAIN GANGLIOSIDES*

1 reference

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Identifiers

10.1038/SJ.MT.6300004

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

ResearchGate publication ID

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