Gene Therapy for Wiskott-Aldrich Syndrome—Long-Term Efficacy and Genotoxicity-Reference-Cited by-同舟云学术

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

Published:2014-03-12 Issue:227 Volume:6 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Braun Christian Jörg¹,Boztug Kaan²,Paruzynski Anna³,Witzel Maximilian¹,Schwarzer Adrian²⁴,Rothe Michael⁴,Modlich Ute⁴,Beier Rita²,Göhring Gudrun⁵,Steinemann Doris⁵,Fronza Raffaele³,Ball Claudia Regina³⁶,Haemmerle Reinhard⁴,Naundorf Sonja⁷,Kühlcke Klaus⁷,Rose Martina⁸,Fraser Chris⁹,Mathias Liesl¹⁰,Ferrari Rudolf¹¹,Abboud Miguel R.¹²,Al-Herz Waleed¹³,Kondratenko Irina¹⁴,Maródi László¹⁵,Glimm Hanno³⁶,Schlegelberger Brigitte⁵,Schambach Axel⁴,Albert Michael Heinrich¹,Schmidt Manfred³,von Kalle Christof³⁶,Klein Christoph¹

Affiliation:

1. Dr. von Hauner Children’s Hospital, Ludwig Maximilians University Munich, 80337 Munich, Germany.

2. Department of Pediatric Hematology/Oncology, Hannover Medical School, 30625 Hannover, Germany.

3. Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, 69120 Heidelberg, Germany.

4. Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.

5. Institute for Cell and Molecular Pathology, Hannover Medical School, 30625 Hannover, Germany.

6. German Cancer Consortium.

7. EUFETS AG, 55743 Idar-Oberstein, Germany.

8. Department of Pediatrics, Minden Hospital, 32429 Minden, Germany.

9. Queensland Children’s Cancer Centre, Royal Children’s Hospital, Brisbane 4006, Queensland, Australia.

10. Department of Pediatric Hematology and Oncology, Loma Linda University Medical Center, Loma Linda, CA 92354, USA.

11. Children’s Hospital Kemperhof, 56073 Koblenz, Germany.

12. Department of Pediatrics and Adolescent Medicine, American University of Beirut Medical Center, Beirut 1107 2020, Lebanon.

13. Department of Pediatrics, Faculty of Medicine, Kuwait University, Kuwait City, Kuwait.

14. Department of Clinical Immunology, Russian Clinical Children’s Hospital, 117513 Moscow, Russia.

15. Department of Infectious and Pediatric Immunology, Medical and Health Science Center, University of Debrecen, 4032 Debrecen, Hungary.

Abstract

Wiskott-Aldrich syndrome gene therapy is feasible, but γ-retroviral vectors contribute a substantial risk of leukemogenesis.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference59 articles.

1. Isolation of a novel gene mutated in Wiskott-Aldrich syndrome

2. Wiskott-Aldrich syndrome

3. Lessons from the Wiskott–Aldrich Syndrome

4. Wasp in immune-system organization and function

5. Wiskott–Aldrich Syndrome: Immunodeficiency resulting from defective cell migration and impaired immunostimulatory activation

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