CNS-targeted AAV5 gene transfer results in global dispersal of vector and prevention of morphological and function deterioration in CNS of globoid cell leukodystrophy mouse model (Q45870410)

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scientific article published on 12 May 2011

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English	CNS-targeted AAV5 gene transfer results in global dispersal of vector and prevention of morphological and function deterioration in CNS of globoid cell leukodystrophy mouse model	scientific article published on 12 May 2011

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scholarly article

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/21620749

CNS-targeted AAV5 gene transfer results in global dispersal of vector and prevention of morphological and function deterioration in CNS of globoid cell leukodystrophy mouse model (English)

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/21620749

Chung-Der Hsiao

2

object named as

Chung-Der Hsiao

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17 December 2017

http://europepmc.org/abstract/MED/21620749

author name string

Dar-Shong Lin

1

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http://europepmc.org/abstract/MED/21620749

Ian Liau

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http://europepmc.org/abstract/MED/21620749

Shuan-Pei Lin

4

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http://europepmc.org/abstract/MED/21620749

Ming-Fu Chiang

5

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http://europepmc.org/abstract/MED/21620749

Chih-Kuang Chuang

6

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http://europepmc.org/abstract/MED/21620749

Tuen-Jen Wang

7

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http://europepmc.org/abstract/MED/21620749

Tsu-Yen Wu

8

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http://europepmc.org/abstract/MED/21620749

Yuan-Ren Jian

9

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http://europepmc.org/abstract/MED/21620749

Sung-Fa Huang

10

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http://europepmc.org/abstract/MED/21620749

Hsuan-Liang Liu

11

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http://europepmc.org/abstract/MED/21620749

publication date

12 May 2011

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http://europepmc.org/abstract/MED/21620749

Molecular Genetics and Metabolism

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http://europepmc.org/abstract/MED/21620749

103

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367-377

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17 December 2017

http://europepmc.org/abstract/MED/21620749

Globoid cell leucodystrophy (Krabbe's disease): deficiency of galactocerebroside beta-galactosidase

1 reference

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Molecular genetics of Krabbe disease (globoid cell leukodystrophy): Diagnostic and clinical implications

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Murine model of genetic demyelinating disease: the twitcher mouse

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The long-term outcomes of presymptomatic infants transplanted for Krabbe disease: report of the workshop held on July 11 and 12, 2008, Holiday Valley, New York

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Gene therapy for lysosomal storage diseases.

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Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements

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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

1 reference

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AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.

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

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Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy

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Combined hematopoietic and lentiviral gene-transfer therapies in newborn Twitcher mice reveal contemporaneous neurodegeneration and demyelination in Krabbe disease

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Molecular cloning and expression of cDNA for murine galactocerebrosidase and mutation analysis of the twitcher mouse, a model of Krabbe's disease

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Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy

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Analysis of galactocerebrosidase activity in the mouse brain by a new histological staining method.

1 reference

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7 January 2021

Pathological and biochemical studies of fetal Krabbe disease

1 reference

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7 January 2021

Neuropathology of twitcher mice: examination by histochemistry, immunohistochemistry, lectin histochemistry and Fourier transform infrared microspectroscopy.

1 reference

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7 January 2021

Spacio-temporal progression of demyelination in twitcher mouse: with clinico-pathological correlation

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7 January 2021

Disorders in myelination in the twitcher mutant: immunohistochemical and biochemical studies.

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

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7 January 2021

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

1 reference

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7 January 2021

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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7 January 2021

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

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Retrograde infection of precerebellar nuclei neurons by injection of a recombinant adenovirus into the cerebellar cortex of normal and reeler mice.

1 reference

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7 January 2021

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

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Widespread correction of lysosomal storage in the mucopolysaccharidosis type VII mouse brain with a herpes simplex virus type 1 vector expressing beta-glucuronidase

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7 January 2021

Cerebellar input to the posterior parietal cortex in the rat.

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Multiple cerebellar zones are involved in the control of individual muscles: a retrograde transneuronal tracing study with rabies virus in the rat.

1 reference

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7 January 2021

Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis

1 reference

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7 January 2021

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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7 January 2021

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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Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate.

1 reference

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Neurodegeneration augments the ability of bone marrow-derived mesenchymal stem cells to fuse with Purkinje neurons in Niemann-Pick type C mice

1 reference

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Lentivector-mediated rescue from cerebellar ataxia in a mouse model of spinocerebellar ataxia.

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Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy.

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Significant correction of pathology in brains of twitcher mice following injection of genetically modified mouse neural progenitor cells

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Retrovirus-mediated gene transfer and galactocerebrosidase uptake into twitcher glial cells results in appropriate localization and phenotype correction

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Storage solutions: treating lysosomal disorders of the brain

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

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1 reference

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Autonomic denervation of lymphoid organs leads to epigenetic immune atrophy in a mouse model of Krabbe disease.

1 reference

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Psychosine accumulates in membrane microdomains in the brain of krabbe patients, disrupting the raft architecture.

1 reference

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Identifiers

10.1016/J.YMGME.2011.05.005

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/21620749

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/21620749

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