Lentivector transduction improves outcomes over transplantation of human HSCs alone in NOD/SCID/Fabry mice. (Q36087322)

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27 January 2020

title

Lentivector transduction improves outcomes over transplantation of human HSCs alone in NOD/SCID/Fabry mice (English)

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27 January 2020

1

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27 January 2020

author name string

Makoto Yoshimitsu

2

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27 January 2020

Nobuo Mizue

3

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27 January 2020

Bryan C Y Au

4

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27 January 2020

James C M Wang

5

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27 January 2020

Xin Fan

6

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27 January 2020

Toshihiro Takenaka

7

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27 January 2020

Jeffrey A Medin

8

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publication date

3 April 2012

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27 January 2020

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27 January 2020

volume

20

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27 January 2020

issue

7

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27 January 2020

page(s)

1454-1461

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Enzyme replacement therapy in Fabry disease: influence on cardiac manifestations.

27 January 2020

cites work

1 reference

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In vivo delivery of human acid ceramidase via cord blood transplantation and direct injection of lentivirus as novel treatment approaches for Farber disease

2 October 2017

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Treatment of lysosomal storage disorders : progress with enzyme replacement therapy

2 October 2017

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Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing.

2 October 2017

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Clinical results of enzyme replacement therapy in Fabry disease: a comprehensive review of literature

2 October 2017

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Neuropathy and Fabry disease: pathogenesis and enzyme replacement therapy

2 October 2017

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

2 October 2017

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Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males

2 October 2017

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Preselective gene therapy for Fabry disease

2 October 2017

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IRES-dependent second gene expression is significantly lower than cap-dependent first gene expression in a bicistronic vector

2 October 2017

1 reference

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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

2 October 2017

1 reference

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Aging accentuates and bone marrow transplantation ameliorates metabolic defects in Fabry disease mice

2 October 2017

1 reference

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

2 October 2017

1 reference

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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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393855

Kidney function and 24-hour proteinuria in patients with Fabry disease during 36 months of agalsidase alfa enzyme replacement therapy: a Brazilian experience.

2 October 2017

1 reference

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Cytokine-pretreatment of CD34(+) cord blood stem cells in vitro reduces long-term cell engraftment in NOD/SCID mice.

23 September 2018

1 reference

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Enzyme replacement therapy in orphan and ultra-orphan diseases: the limitations of standard economic metrics as exemplified by Fabry-Anderson disease.

23 September 2018

1 reference

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

23 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393855

Engraftment of human CD34+ cells leads to widespread distribution of donor-derived cells and correction of tissue pathology in a novel murine xenotransplantation model of lysosomal storage disease.

23 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393855

Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 60 obligate carrier females

23 September 2018

1 reference

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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.

23 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3393855

Enzymatic Defect in Fabry's Disease

23 September 2018

1 reference

12 December 2020

Comparison of repopulating ability of hematopoietic progenitor cells isolated from human umbilical cord blood or bone marrow cells in NOD/SCID mice

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12 December 2020

Human short-term repopulating stem cells are efficiently detected following intrafemoral transplantation into NOD/SCID recipients depleted of CD122+ cells

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12 December 2020

Clinical benefit of enzyme replacement therapy in Fabry disease

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12 December 2020

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12 December 2020

Identifiers

DOI

10.1038/MT.2012.64

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22472949%20AND%20SRC:MED&resulttype=core&format=json

27 January 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22472949%20AND%20SRC:MED&resulttype=core&format=json

27 January 2020

1 reference

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