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Braun CJ, Witzel M, Paruzynski A, et al. Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis. Rare Dis. 2014;2(1):e947749doi: 10.4161/21675511.2014.947749.
Braun, C. J., Witzel, M., Paruzynski, A., Boztug, K., von Kalle, C., Schmidt, M., & Klein, C. (2014). Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis. Rare diseases (Austin, Tex.), 2(1), e947749. https://doi.org/10.4161/21675511.2014.947749
Braun, Christian Joerg, et al. "Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis." Rare diseases (Austin, Tex.) vol. 2,1 (2014): e947749. doi: https://doi.org/10.4161/21675511.2014.947749
Braun CJ, Witzel M, Paruzynski A, Boztug K, von Kalle C, Schmidt M, Klein C. Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis. Rare Dis. 2014 Oct 30;2(1):e947749. doi: 10.4161/21675511.2014.947749. eCollection 2014. PMID: 26942098; PMCID: PMC4755244.
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Rare Dis. 2014 Oct 30;2(1):e947749. doi: 10.4161/21675511.2014.947749. eCollection 2014.

Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis.

Rare diseases (Austin, Tex.)

Christian Joerg Braun, Maximilian Witzel, Anna Paruzynski, Kaan Boztug, Christof von Kalle, Manfred Schmidt, Christoph Klein

Affiliations

  1. Dr. von Hauner Children's Hospital; Ludwig Maximilians University Munich ; Munich, Germany.
  2. Department of Translational Oncology; National Center for Tumor Diseases and German Cancer Research Center; Heidelberg, Germany; New address: BioNTech AG; Mainz, Germany.
  3. Department of Pediatric Hematology/Oncology; Hannover Medical School; Hannover, Germany; Present address: CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences/Department of Pediatrics and Adolescent Medicine, Medical University of Vienna; Vienna, Austria.
  4. Department of Translational Oncology; National Center for Tumor Diseases and German Cancer Research Center ; Heidelberg, Germany.

PMID: 26942098 PMCID: PMC4755244 DOI: 10.4161/21675511.2014.947749

Abstract

Wiskott-Aldrich-Syndrome (WAS) is a rare X-linked recessive disease caused by mutations of the WAS gene. It is characterized by immunodeficiency, autoimmunity, low numbers of small platelets (microthrombocytopenia) and a high risk of cancer, especially B cell lymphoma and leukemia.

Keywords: Wiskott-Aldrich-Syndrome; gene therapy; immunodeficiency; insertional mutagenesis; leukemia

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