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

@article{Ott2006CorrectionOX,
  title={Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1},
  author={Marion Gabriele Ott and Manfred Schmidt and Kerstin Schwarzwaelder and Stefan Stein and Ulrich Siler and Ulrike Koehl and Hanno Glimm and Klaus Kühlcke and Andrea J Schilz and Hana Kunkel and Sonja Naundorf and Andrea Brinkmann and Annette Deichmann and Marlene Fischer and Claudia R. Ball and Ingo H. Pilz and Cynthia E. Dunbar and Yang Du and Nancy A. Jenkins and Neal G. Copeland and Ursula Lüthi and Moustapha Hassan and Adrian J. Thrasher and Dieter Hoelzer and Christof von Kalle and Reinhard A. Seger and Manuel Grez},
  journal={Nature Medicine},
  year={2006},
  volume={12},
  pages={401-409}
}
Gene transfer into hematopoietic stem cells has been used successfully for correcting lymphoid but not myeloid immunodeficiencies. Here we report on two adults who received gene therapy after nonmyeloablative bone marrow conditioning for the treatment of X-linked chronic granulomatous disease (X-CGD), a primary immunodeficiency caused by a defect in the oxidative antimicrobial activity of phagocytes resulting from mutations in gp91phox. We detected substantial gene transfer in both individuals… 

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...

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