Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy (Q33649318)

28 July 2017

Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy (English)

1 reference

28 July 2017

0 references

5

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2 June 2020

Luigi Naldini

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2 June 2020

Eugenio Montini

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2 June 2020

Sabata Martino

9

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2 June 2020

Angela Gritti

10

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2 June 2020

1

Annalisa Lattanzi

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2 June 2020

3

Claudio Maderna

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2 June 2020

6

Willem Kulik

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2 June 2020

author name string

Camilla Salvagno

2

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2 June 2020

Fabrizio Benedicenti

4

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2 June 2020

publication date

23 January 2014

1 reference

28 July 2017

19

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inferred from page(s)

published in

Human Molecular Genetics

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2 June 2020

volume

23

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2 June 2020

issue

12

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2 June 2020

page(s)

3250-3268

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describes a project that uses

2 June 2020

ImageJ

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11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC4030779/fullTextXML

inferred from PubMed Central ID database lookup

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21 June 2018

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21 June 2018

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Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy

21 June 2018

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Corticospinal tract transduction: a comparison of seven adeno-associated viral vector serotypes and a non-integrating lentiviral vector

21 June 2018

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Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

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21 June 2018

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21 June 2018

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21 June 2018

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Single-dose intracerebroventricular administration of galactocerebrosidase improves survival in a mouse model of globoid cell leukodystrophy

21 June 2018

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21 June 2018

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21 June 2018

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21 June 2018

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Expression and purification of a human, soluble Arylsulfatase A for Metachromatic Leukodystrophy enzyme replacement therapy

21 June 2018

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21 June 2018

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21 June 2018

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Krabbe disease: genetic aspects and progress toward therapy

21 June 2018

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21 June 2018

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Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy

21 June 2018

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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

21 June 2018

1 reference

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The twitcher mouse: a model for Krabbe disease and for experimental therapies

21 June 2018

1 reference

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Progressive accumulation of toxic metabolite in a genetic leukodystrophy

21 June 2018

1 reference

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Histochemical similarities between human and animal globoid cells in Krabbe's disease: a lectin study.

21 June 2018

1 reference

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The galactocerebrosidase enzyme contributes to maintain a functional neurogenic niche during early post-natal CNS development

21 June 2018

1 reference

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Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

30 October 2018

1 reference

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Axonopathy is a compounding factor in the pathogenesis of Krabbe disease.

30 October 2018

1 reference

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Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy

30 October 2018

1 reference

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

30 October 2018

1 reference

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

30 October 2018

1 reference

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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

30 October 2018

1 reference

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Specific determination of beta-galactocerebrosidase activity via competitive inhibition of beta-galactosidase

30 October 2018

1 reference

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

30 October 2018

1 reference

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High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR).

30 October 2018

1 reference

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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

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4030779

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

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4030779

Immune responses to AAV in a phase I study for Canavan disease.

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4030779

Characterization of the expression of the ALK receptor tyrosine kinase in 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=4030779

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

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4030779

Hematopoietic cell transplantation ameliorates clinical phenotype and progression of the CNS pathology in the mouse model of late onset Krabbe disease

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4030779

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

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4030779

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

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4030779

Lentiviral gene transfer to the nonhuman primate brain

30 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4030779

New genes involved in cancer identified by retroviral tagging

30 October 2018

1 reference

12 December 2020

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

1 reference

12 December 2020

Identification, isolation, and promoter-defined separation of mitotic oligodendrocyte progenitor cells from the adult human subcortical white matter

1 reference

12 December 2020

Gene therapy for lysosomal storage disorders

1 reference

12 December 2020

Identifiers

DOI

10.1093/HMG/DDU034

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24463623%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24463623%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24463623%20AND%20SRC:MED&resulttype=core&format=json