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

Gene-modified autologous hematopoietic stem cells (HSC) can provide ample clinical benefits to subjects suffering from X-linked chronic granulomatous disease (X-CGD), a rare inherited immunodeficiency characterized by recurrent, often life-threatening bacterial and fungal infections. Here we report...

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Main Authors:	Stein, Stefan (Author) , Ott, Marion G. (Author) , Schultze-Straßer, Stephan (Author) , Jauch, Anna (Author) , Burwinkel, Barbara (Author) , Kinner, Andrea (Author) , Schmidt, Manfred (Author) , Krämer, Alwin (Author) , Schwäble, Joachim (Author) , Glimm, Hanno (Author) , Köhl, Ulrike (Author) , Preiss, Carolin (Author) , Ball, Claudia R. (Author) , Martin, Hans (Author) , Göhring, Gudrun (Author) , Schwarzwälder, Kerstin (Author) , Hofmann, Wolf-Karsten (Author) , Karakaya, Kadin (Author) , Tchatchou, Sandrine (Author) , Yang, Rongxi (Author) , Reinecke, Petra (Author) , Kühlcke, Klaus (Author) , Schlegelberger, Brigitte (Author) , Thrasher, Adrian J. (Author) , Hoelzer, Dieter (Author) , Seger, Reinhard (Author) , Kalle, Christof von (Author) , Grez, Manuel (Author)
Format:	Article (Journal)
Language:	English
Published:	24 January 2010
In:	Nature medicine Year: 2010, Volume: 16, Issue: 2, Pages: 198-204
ISSN:	1546-170X
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Author Notes:	Stefan Stein, Marion G. Ott, Stephan Schultze-Strasser, Anna Jauch, Barbara Burwinkel, Andrea Kinner, Manfred Schmidt, Alwin Krämer, Joachim Schwäble, Hanno Glimm, Ulrike Koehl, Carolin Preiss, Claudia Ball, Hans Martin, Gudrun Göhring, Kerstin Schwarzwaelder, Wolf-Karsten Hofmann, Kadin Karakaya, Sandrine Tchatchou, Rongxi Yang, Petra Reinecke, Klaus Kühlcke, Brigitte Schlegelberger, Adrian J. Thrasher, Dieter Hoelzer, Reinhard Seger, Christof von Kalle & Manuel Grez