Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity (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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Anna Paruzynski

3

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Maximilian Witzel

4

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Adrian Schwarzer

5

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Michael Rothe

6

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Ute Modlich

7

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Rita Beier

8

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Doris Steinemann

10

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Raffaele Fronza

11

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Reinhard Haemmerle

13

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Sonja Naundorf

14

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Klaus Kühlcke

15

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Martina Rose

16

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Chris Fraser

17

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Liesl Mathias

18

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Rudolf Ferrari

19

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Miguel R Abboud

20

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Waleed Al-Herz

21

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Irina Kondratenko

22

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László Maródi

23

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Hanno Glimm

24

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Michael Heinrich Albert

27

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

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

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A census of human cancer genes

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

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21 January 2018

Identifiers

DOI

1 reference

26 July 2017

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