Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors (Q33408529)

26 July 2017

title

Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors (English)

1 reference

26 July 2017

main subject

Wiskott-Aldrich syndrome

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author name string

Rachel M Koldej

1

1 reference

26 July 2017

Gael Carney

2

1 reference

26 July 2017

Matthew M Wielgosz

3

1 reference

26 July 2017

Sheng Zhou

4

1 reference

26 July 2017

Jun Zhan

5

1 reference

26 July 2017

Brian P Sorrentino

6

1 reference

26 July 2017

Arthur W Nienhuis

7

1 reference

26 July 2017

publication date

20 June 2013

1 reference

Human gene therapy. Clinical development

26 July 2017

published in

1 reference

26 July 2017

volume

24

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26 July 2017

issue

2

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26 July 2017

page(s)

77-85

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Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome

26 July 2017

cites work

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14 July 2018

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14 July 2018

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Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

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14 July 2018

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14 July 2018

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14 July 2018

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14 July 2018

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Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome

14 July 2018

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14 July 2018

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Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models

14 July 2018

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Optimized lentiviral vector design improves titer and transgene expression of vectors containing the chicken beta-globin locus HS4 insulator element

14 July 2018

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WASP confers selective advantage for specific hematopoietic cell populations and serves a unique role in marginal zone B-cell homeostasis and function

14 July 2018

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Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

14 July 2018

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14 July 2018

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14 July 2018

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Extended core sequences from the cHS4 insulator are necessary for protecting retroviral vectors from silencing position effects

14 July 2018

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14 July 2018

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Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients.

14 July 2018

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14 July 2018

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14 July 2018

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14 July 2018

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14 July 2018

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Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction.

14 July 2018

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14 July 2018

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14 July 2018

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14 July 2018

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14 July 2018

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14 July 2018

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Characterization of the chicken beta-globin insulator

14 July 2018

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A 5' element of the chicken beta-globin domain serves as an insulator in human erythroid cells and protects against position effect in Drosophila

14 July 2018

1 reference

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Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells.

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003467

Outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome.

14 July 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003467

Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction

31 October 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003467

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31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003467

Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation.

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003467

Failure of SCID-X1 gene therapy in older patients

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003467

Functional correction of T cells derived from patients with the Wiskott–Aldrich syndrome (WAS) by transduction with an oncoretroviral vector encoding the WAS protein

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003467

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

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003467

Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003467

Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4003467

Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program

31 October 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23786330

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23786330

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1089/HUMC.2012.244

1 reference

26 July 2017

1 reference

26 July 2017

1 reference