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

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

doi:10.4161/21675511.2014.947749

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.

Authors

Christian Joerg Braun¹, Maximilian Witzel¹, Anna Paruzynski², Kaan Boztug³, Christof von Kalle⁴, Manfred Schmidt⁴, Christoph Klein¹

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.

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.