Stem-cell gene therapy for the Wiskott-Aldrich syndrome (Q33392766)

26 July 2017

title

Stem-cell gene therapy for the Wiskott-Aldrich syndrome (English)

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26 July 2017

main subject

Wiskott-Aldrich syndrome

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Christof von Kalle

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Jordan S. Orange

object named as

Jordan S Orange

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26 July 2017

Ricardo A Dewey

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Claudia R Ball

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Manfred Schmidt

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26 July 2017

Kaan Boztug

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26 July 2017

Adrian Schwarzer

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26 July 2017

Pinaki P Banerjee

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26 July 2017

Inés Avedillo Díez

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26 July 2017

Marie Böhm

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26 July 2017

Ali Nowrouzi

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26 July 2017

Hanno Glimm

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26 July 2017

Sonja Naundorf

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26 July 2017

Klaus Kühlcke

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26 July 2017

Rainer Blasczyk

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26 July 2017

Irina Kondratenko

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26 July 2017

László Maródi

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26 July 2017

Christoph Klein

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26 July 2017

language of work or name

English

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publication date

1 November 2010

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The New England Journal of Medicine

26 July 2017

published in

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26 July 2017

volume

363

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26 July 2017

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26 July 2017

page(s)

1918-1927

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WASP: a key immunological multitasker

26 July 2017

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

21 June 2018

1 reference

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Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

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

21 June 2018

1 reference

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Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

Wiskott-Aldrich syndrome protein is required for regulatory T cell homeostasis

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

Retroviral WASP gene transfer into human hematopoietic stem cells reconstitutes the actin cytoskeleton in myeloid progeny cells differentiated in vitro.

21 June 2018

1 reference

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Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

Deficiency in the Wiskott-Aldrich protein induces premature proplatelet formation and platelet production in the bone marrow compartment

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

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

21 June 2018

1 reference

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Clinical course of patients with WASP gene mutations

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

Wiskott-Aldrich syndrome protein is required for NK cell cytotoxicity and colocalizes with actin to NK cell-activating immunologic synapses

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

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

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

Isolation of a novel gene mutated in Wiskott-Aldrich syndrome

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR).

31 October 2018

1 reference

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Lessons from the Wiskott-Aldrich syndrome.

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

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=3064520

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

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3064520

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=3064520

31 October 2018

Wiskott-Aldrich syndrome

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21067383

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1056/NEJMOA1003548

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26 July 2017

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26 July 2017

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