Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. (Q34508425)

2 August 2017

title

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

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X-linked chronic granulomatous disease

2 August 2017

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Christof von Kalle

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Stefan Stein

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Claudia Ball

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Ulrike Koehl

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Moustapha Hassan

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Cynthia Dunbar

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Adrian J Thrasher

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Neal G Copeland

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2 August 2017

Nancy A Jenkins

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Manfred Schmidt

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Manuel Grez

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2 August 2017

Kerstin Schwarzwaelder

object named as

Kerstin Schwarzwaelder

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2 August 2017

Hanno Glimm

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2 August 2017

Dieter Hoelzer

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2 August 2017

Marion G Ott

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2 August 2017

Ulrich Siler

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2 August 2017

Klaus Kühlcke

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2 August 2017

Andrea Schilz

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2 August 2017

Hana Kunkel

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Sonja Naundorf

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2 August 2017

Andrea Brinkmann

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Annette Deichmann

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Marlene Fischer

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2 August 2017

Ingo Pilz

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2 August 2017

Yang Du

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Ursula Lüthi

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Reinhard Seger

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2 August 2017

language of work or name

English

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publication date

2 April 2006

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2 August 2017

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2 August 2017

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2 August 2017

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2 August 2017

page(s)

401-409

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

2 August 2017

cites work

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

21 January 2018

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

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

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Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease

21 January 2018

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T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.

21 January 2018

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The genetic basis of chronic granulomatous disease

21 January 2018

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The NADPH oxidase and chronic granulomatous disease

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Genetic, biochemical, and clinical features of chronic granulomatous disease.

21 January 2018

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Chronic granulomatous disease.

21 January 2018

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Chronic granulomatous disease. Report on a national registry of 368 patients

21 January 2018

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Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000.

21 January 2018

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Long-term high-level reconstitution of NADPH oxidase activity in murine X-linked chronic granulomatous disease using a bicistronic vector expressing gp91phox and a Delta LNGFR cell surface marker

21 January 2018

1 reference

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

21 January 2018

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Chance or necessity? Insertional mutagenesis in gene therapy and its consequences

21 January 2018

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Stem cell clonality and genotoxicity in hematopoietic cells: gene activation side effects should be avoidable.

21 January 2018

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Polyclonal long-term repopulating stem cell clones in a primate model

21 January 2018

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Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates

21 January 2018

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Location of the epitope for 7D5, a monoclonal antibody raised against human flavocytochrome b558, to the extracellular peptide portion of primate gp91phox

21 January 2018

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Pharmacokinetics and distribution of liposomal busulfan in the rat: a new formulation for intravenous administration

21 January 2018

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Pharmacokinetics of liposomal busulphan in man.

21 January 2018

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Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells

21 January 2018

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Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking.

21 January 2018

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Failure of SCID-X1 gene therapy in older patients

21 January 2018

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EVI1 induces myelodysplastic syndrome in mice

21 January 2018

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Cytogenetic and molecular genetic evolution of chronic myeloid leukemia.

21 January 2018

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The role of EVI1 in normal and leukemic cells

21 January 2018

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Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells

21 January 2018

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Efficient marking of human cells with rapid but transient repopulating activity in autografted recipients

21 January 2018

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Variable correction of host defense following gene transfer and bone marrow transplantation in murine X-linked chronic granulomatous disease

21 January 2018

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X-linked inheritance in females with chronic granulomatous disease

21 January 2018

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Unusual late presentation of X-linked chronic granulomatous disease in an adult female with a somatic mosaic for a novel mutation in CYBB.

21 January 2018

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Gene therapy for chronic granulomatous disease

21 January 2018

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7 January 2021

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7 January 2021

New genes involved in cancer identified by retroviral tagging

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7 January 2021

Kinetic microplate assay for superoxide production by neutrophils and other phagocytic cells

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7 January 2021

Novel retroviral vectors for efficient expression of the multidrug resistance (mdr-1) gene in early hematopoietic cells.

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7 January 2021

Murine leukemia induced by retroviral gene marking

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7 January 2021

Recurrent severe infections in a girl with apparently variable expression of mosaicism for chronic granulomatous disease

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7 January 2021

Increased susceptibility of a carrier of X-linked chronic granulomatous disease (CGD) to Aspergillus fumigatus infection associated with age-related skewing of lyonization

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7 January 2021

Identifiers

DOI

10.1038/NM1393

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2 August 2017

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