Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer (Q34161415)

31 July 2017

Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer (English)

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31 July 2017

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Hiroshi Takahashi

1

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31 July 2017

Yukihiko Hirai

2

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31 July 2017

Makoto Migita

3

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31 July 2017

Yoshihiko Seino

4

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31 July 2017

Yuh Fukuda

5

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31 July 2017

Hitoshi Sakuraba

6

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31 July 2017

Ryoichi Kase

7

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31 July 2017

Toshihide Kobayashi

8

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31 July 2017

Yasuhiro Hashimoto

9

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31 July 2017

Takashi Shimada

10

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31 July 2017

language of work or name

English

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publication date

7 October 2002

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Proceedings of the National Academy of Sciences of the United States of America

31 July 2017

6

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based on heuristic

inferred from page(s)

published in

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31 July 2017

99

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31 July 2017

21

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31 July 2017

13777-13782

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

31 July 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

A journey to the world of glycobiology

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Dose response to a single intramuscular injection of recombinant adeno-associated virus-erythropoietin in monkeys

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Persistent expression of canine factor IX in hemophilia B canines

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

alpha-Galactosidase A deficient mice: a model of Fabry disease

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Generation of one set of murine monoclonal antibodies specific for globo-series glycolipids: evidence for differential distribution of the glycolipids in rat small intestine

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Immunofluorescence imaging diagnosis of Fabry heterozygotes using confocal laser scanning microscopy.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Adeno-associated virus vectors preferentially transduce cells in S phase.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Lysosomal enzymes and their receptors

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Site-directed mutagenesis by gene targeting in mouse embryo-derived stem cells

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Expression vector system based on the chicken beta-actin promoter directs efficient production of interleukin-5.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Indiscriminate activity from the B19 parvovirus p6 promoter in nonpermissive cells

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Purification and properties of the two major isozymes of alpha-galactosidase from human placenta

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

5 July 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.222221899

A new strategy for large-scale preparation of high-titer recombinant adeno-associated virus vectors by using packaging cell lines and sulfonated cellulose column chromatography

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.222221899

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.222221899

Recombinant adeno-associated virus for muscle directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.222221899

Improvement of erythropoiesis in beta-thalassemic mice by continuous erythropoietin delivery from muscle

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=129774

Enzymatic Defect in Fabry's Disease

28 October 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.222221899

An atypical variant of Fabry's disease in men with left ventricular hypertrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.222221899

Replacement Therapy for Inherited Enzyme Deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.222221899

Differential assay for lysosomal alpha-galactosidases in human tissues and its application to Fabry's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.222221899

Organ-specific difference in the sugar chains of gamma-glutamyltranspeptidase

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.222221899

21 January 2018

Identifiers

DOI

10.1073/PNAS.222221899

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31 July 2017

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31 July 2017

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