Preselective gene therapy for Fabry disease (Q34736973)

4 August 2017

title

Preselective gene therapy for Fabry disease (English)

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4 August 2017

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Qin G

1

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4 August 2017

Takenaka T

2

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4 August 2017

Telsch K

3

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4 August 2017

Kelley L

4

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4 August 2017

Howard T

5

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4 August 2017

Levade T

6

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4 August 2017

Deans R

7

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4 August 2017

Howard BH

8

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4 August 2017

Malech HL

9

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4 August 2017

Brady RO

10

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4 August 2017

Medin JA

11

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4 August 2017

language of work or name

English

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publication date

6 March 2001

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

4 August 2017

published in

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4 August 2017

volume

98

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4 August 2017

issue

6

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4 August 2017

page(s)

3428-3433

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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

4 August 2017

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Preselection of retrovirally transduced bone marrow avoids subsequent stem cell gene silencing and age-dependent extinction of expression of human beta-globin in engrafted mice.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Ex vivo expansion and genetic marking of primitive human and baboon hematopoietic cells

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: improved retroviral vectors for clinical trials

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Model SV40-transformed fibroblast lines for metabolic studies of human prosaposin and acid ceramidase deficiencies

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Selection of retrovirally transduced cells to enhance the efficiency of gene therapy.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

A bicistronic therapeutic retroviral vector enables sorting of transduced CD34+ cells and corrects the enzyme deficiency in cells from Gaucher patients.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Rapid and efficient selection of human hematopoietic cells expressing murine heat-stable antigen as an indicator of retroviral-mediated gene transfer

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Endocytosis of lysosomal alpha-galactosidase A by cultured fibroblasts from patients with Fabry disease

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Retrovirus-mediated transfer and expression of drug resistance genes in human haematopoietic progenitor cells

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

A safe packaging line for gene transfer: separating viral genes on two different plasmids

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Construction and properties of retrovirus packaging cells based on gibbon ape leukemia virus

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Targeting of proteins to the lysosome.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Nectadrin, the heat-stable antigen, is a cell adhesion molecule

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Heat-stable antigen is a costimulatory molecule for CD4 T cell growth

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

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.

7 July 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.061020598

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.061020598

T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.061020598

In vivo methotrexate selection of murine hemopoietic cells transduced with a retroviral vector for Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.061020598

Long-term cure of the photosensitivity of murine erythropoietic protoporphyria by preselective gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.061020598

Immune response to green fluorescent protein: implications for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.061020598

HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.061020598

Detection and isolation of gene-corrected cells in Gaucher disease via a fluorescence-activated cell sorter assay for lysosomal glucocerebrosidase activity.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Selection of retrovirally transduced hematopoietic cells using CD24 as a marker of gene transfer.

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=30670

Genes with promoters in retrovirus vectors can be independently suppressed by an epigenetic mechanism

26 October 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/11248095

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Transduction of murine bone marrow cells with an MDR1 vector enables ex vivo stem cell expansion, but these expanded grafts cause a myeloproliferative syndrome in transplanted mice

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/11248095

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1073/PNAS.061020598

1 reference

4 August 2017

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4 August 2017

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