(Q45861979)

17 December 2017

title

Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells (English)

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17 December 2017

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Yoshimitsu M

1

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17 December 2017

Higuchi K

2

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17 December 2017

Ramsubir S

3

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17 December 2017

Nonaka T

4

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17 December 2017

Rasaiah VI

5

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17 December 2017

Siatskas C

6

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17 December 2017

Liang SB

7

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17 December 2017

Murray GJ

8

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17 December 2017

Brady RO

9

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17 December 2017

Medin JA

10

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17 December 2017

publication date

24 August 2006

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17 December 2017

published in

Gene Therapy

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17 December 2017

volume

14

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17 December 2017

issue

3

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17 December 2017

page(s)

256-265

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17 December 2017

https://scigraph.springernature.com/pub.10.1038/sj.gt.3302839

exact match

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cites work

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Enzyme replacement therapy in Fabry disease: a randomized controlled trial

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

21 January 2018

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Cord-blood transplants from unrelated donors in patients with Hurler's syndrome

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Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999.

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Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and S

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Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

21 January 2018

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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

21 January 2018

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Circulating alpha-galactosidase A derived from transduced bone marrow cells: relevance for corrective gene transfer for Fabry disease.

21 January 2018

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

21 January 2018

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Preselective gene therapy for Fabry disease

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Lentiviruses as gene transfer agents for delivery to non-dividing cells

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Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system

21 January 2018

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Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors

21 January 2018

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

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

21 January 2018

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

21 January 2018

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Retrovirus-mediated correction of the metabolic defect in cultured Farber disease cells.

21 January 2018

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Enzymatic and functional correction along with long-term enzyme secretion from transduced bone marrow hematopoietic stem/progenitor and stromal cells derived from patients with Fabry disease.

21 January 2018

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

21 January 2018

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Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors

21 January 2018

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Immunohistochemical characterization of transgenic mice highly expressing human lysosomal alpha-galactosidase.

21 January 2018

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Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy

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Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

21 January 2018

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Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

21 January 2018

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A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

21 January 2018

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LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

21 January 2018

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HIV-1 integration in the human genome favors active genes and local hotspots

21 January 2018

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Transcription start regions in the human genome are favored targets for MLV integration

21 January 2018

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Expression and secretion of human glucocerebrosidase mediated by recombinant lentivirus vectors in vitro and in vivo: implications for gene therapy of Gaucher disease

21 January 2018

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Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease.

21 January 2018

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Enzymatic Defect in Fabry's Disease

21 January 2018

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7 January 2021

An Atypical Variant of Fabry's Disease in Men with Left Ventricular Hypertrophy

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302839

7 January 2021

Transduction of Human CD34+CD38- Bone Marrow and Cord Blood-Derived SCID-Repopulating Cells with Third-Generation Lentiviral Vectors

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302839

7 January 2021

Specific pharmacological dimerization of KDR in lentivirally transduced human hematopoietic cells activates anti-apoptotic and proliferative mechanisms

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302839

7 January 2021

Murine leukemia induced by retroviral gene marking

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302839

7 January 2021

AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302839

7 January 2021

Identifiers

DOI

10.1038/SJ.GT.3302839

1 reference

17 December 2017

Dimensions Publication ID

0 references

1 reference

17 December 2017