title: Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis.
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.
authors.affiliation: Dr. von Hauner Children's Hospital; Ludwig Maximilians University Munich ; Munich, Germany.
authors.forename: Christian Joerg
authors.initials: CJ
authors.lastname: Braun
authors.affiliation: Dr. von Hauner Children's Hospital; Ludwig Maximilians University Munich ; Munich, Germany.
authors.forename: Maximilian
authors.initials: M
authors.lastname: Witzel
authors.affiliation: Department of Translational Oncology; National Center for Tumor Diseases and German Cancer Research Center; Heidelberg, Germany; New address: BioNTech AG; Mainz, Germany.
authors.forename: Anna
authors.initials: A
authors.lastname: Paruzynski
authors.affiliation: 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.
authors.forename: Kaan
authors.initials: K
authors.lastname: Boztug
authors.affiliation: Department of Translational Oncology; National Center for Tumor Diseases and German Cancer Research Center ; Heidelberg, Germany.
authors.forename: Christof
authors.initials: C
authors.lastname: von Kalle
authors.affiliation: Department of Translational Oncology; National Center for Tumor Diseases and German Cancer Research Center ; Heidelberg, Germany.
authors.forename: Manfred
authors.initials: M
authors.lastname: Schmidt
authors.affiliation: Dr. von Hauner Children's Hospital; Ludwig Maximilians University Munich ; Munich, Germany.
authors.forename: Christoph
authors.initials: C
authors.lastname: Klein
keywords: Wiskott-Aldrich-Syndrome; gene therapy; immunodeficiency; insertional mutagenesis; leukemia