Research ArticleGene Therapy

Gene Therapy for Wiskott-Aldrich Syndrome—Long-Term Efficacy and Genotoxicity

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Science Translational Medicine  12 Mar 2014:
Vol. 6, Issue 227, pp. 227ra33
DOI: 10.1126/scitranslmed.3007280

Article Information

vol. 6 no. 227 227ra33

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication August 8, 2013
  • Accepted for publication January 28, 2014
  • .

Author Information

  1. Christian Jörg Braun1,*,
  2. Kaan Boztug2,*,,
  3. Anna Paruzynski3,*,
  4. Maximilian Witzel1,*,
  5. Adrian Schwarzer2,4,
  6. Michael Rothe4,
  7. Ute Modlich4,
  8. Rita Beier2,
  9. Gudrun Göhring5,
  10. Doris Steinemann5,
  11. Raffaele Fronza3,
  12. Claudia Regina Ball3,6,
  13. Reinhard Haemmerle4,
  14. Sonja Naundorf7,
  15. Klaus Kühlcke7,
  16. Martina Rose8,
  17. Chris Fraser9,
  18. Liesl Mathias10,
  19. Rudolf Ferrari11,
  20. Miguel R. Abboud12,
  21. Waleed Al-Herz13,
  22. Irina Kondratenko14,
  23. László Maródi15,
  24. Hanno Glimm3,6,
  25. Brigitte Schlegelberger5,
  26. Axel Schambach4,
  27. Michael Heinrich Albert1,
  28. Manfred Schmidt3,*,
  29. Christof von Kalle3,6,* and
  30. Christoph Klein1,*,
  1. 1Dr. von Hauner Children’s Hospital, Ludwig Maximilians University Munich, 80337 Munich, Germany.
  2. 2Department of Pediatric Hematology/Oncology, Hannover Medical School, 30625 Hannover, Germany.
  3. 3Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, 69120 Heidelberg, Germany.
  4. 4Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.
  5. 5Institute for Cell and Molecular Pathology, Hannover Medical School, 30625 Hannover, Germany.
  6. 6German Cancer Consortium.
  7. 7EUFETS AG, 55743 Idar-Oberstein, Germany.
  8. 8Department of Pediatrics, Minden Hospital, 32429 Minden, Germany.
  9. 9Queensland Children’s Cancer Centre, Royal Children’s Hospital, Brisbane 4006, Queensland, Australia.
  10. 10Department of Pediatric Hematology and Oncology, Loma Linda University Medical Center, Loma Linda, CA 92354, USA.
  11. 11Children’s Hospital Kemperhof, 56073 Koblenz, Germany.
  12. 12Department of Pediatrics and Adolescent Medicine, American University of Beirut Medical Center, Beirut 1107 2020, Lebanon.
  13. 13Department of Pediatrics, Faculty of Medicine, Kuwait University, Kuwait City, Kuwait.
  14. 14Department of Clinical Immunology, Russian Clinical Children’s Hospital, 117513 Moscow, Russia.
  15. 15Department of Infectious and Pediatric Immunology, Medical and Health Science Center, University of Debrecen, 4032 Debrecen, Hungary.
  1. Corresponding author. E-mail: christoph.klein{at}med.uni-muenchen.de
  • * These authors contributed equally to this work and should be considered aequo loco.

  • Present address: CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences/Department of Paediatrics and Adolescent Medicine, Medical University of Vienna, Lazarettgasse 14 AKH BT 25.3, A-1090 Vienna, Austria.

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