(Q33413888)

26 July 2017

title

Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity (English)

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26 July 2017

main subject

Wiskott-Aldrich syndrome

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29

Christof von Kalle

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Axel Schambach

26

Axel Schambach

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Brigitte Schlegelberger

25

Brigitte Schlegelberger

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Christian J Braun

Christian Jörg Braun

1

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Claudia R Ball

Claudia Regina Ball

12

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Manfred Schmidt

28

Manfred Schmidt

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26 July 2017

9

Gudrun Göhring

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26 July 2017

Kaan Boztug

2

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26 July 2017

Anna Paruzynski

3

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26 July 2017

Maximilian Witzel

4

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26 July 2017

Adrian Schwarzer

5

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26 July 2017

Michael Rothe

6

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26 July 2017

Ute Modlich

7

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26 July 2017

Rita Beier

8

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26 July 2017

Doris Steinemann

10

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26 July 2017

Raffaele Fronza

11

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26 July 2017

Reinhard Haemmerle

13

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26 July 2017

Sonja Naundorf

14

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26 July 2017

Klaus Kühlcke

15

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26 July 2017

Martina Rose

16

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26 July 2017

Chris Fraser

17

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26 July 2017

Liesl Mathias

18

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26 July 2017

Rudolf Ferrari

19

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26 July 2017

Miguel R Abboud

20

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26 July 2017

Waleed Al-Herz

21

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26 July 2017

Irina Kondratenko

22

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26 July 2017

László Maródi

23

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26 July 2017

Hanno Glimm

24

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26 July 2017

Michael Heinrich Albert

27

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26 July 2017

Christoph Klein

30

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26 July 2017

language of work or name

English

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publication date

1 March 2014

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Science Translational Medicine

26 July 2017

published in

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26 July 2017

volume

6

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26 July 2017

issue

227

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26 July 2017

page(s)

227ra33

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Isolation of a novel gene mutated in Wiskott-Aldrich syndrome

26 July 2017

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Wiskott-Aldrich Syndrome: Immunodeficiency resulting from defective cell migration and impaired immunostimulatory activation

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

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

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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Insertional mutagenesis in gene therapy and stem cell biology

21 January 2018

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Stem-cell gene therapy for the Wiskott-Aldrich syndrome

21 January 2018

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Analysis of T-cell repertoire diversity in Wiskott-Aldrich syndrome

21 January 2018

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Cdc42-interacting protein-4 functionally links actin and microtubule networks at the cytolytic NK cell immunological synapse.

21 January 2018

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

21 January 2018

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A census of human cancer genes

21 January 2018

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

21 January 2018

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21 January 2018

Measurement of Diversity

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Multiple mechanisms induce ectopic expression of LYL1 in subsets of T-ALL cell lines

21 January 2018

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Late MRD response determines relapse risk overall and in subsets of childhood T-cell ALL: results of the AIEOP-BFM-ALL 2000 study.

21 January 2018

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MN1 overexpression is an important step in the development of inv(16) AML.

21 January 2018

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Failure of SCID-X1 gene therapy in older patients

21 January 2018

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Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo

21 January 2018

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Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy

21 January 2018

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Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy

21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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Activation of the T-cell oncogene LMO2 after gene therapy for X-linked severe combined immunodeficiency

21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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10.1126/SCITRANSLMED.3007280

21 January 2018

Identifiers

DOI

1 reference

26 July 2017

1 reference