Cellular Therapies for Wiskott-Aldrich Syndrome

  • Christian J. Braun
  • Christoph KleinEmail author


Wiskott-Aldrich syndrome (WAS) is an X-linked inherited rare disease caused by mutations of the WAS gene and affecting various subsets of the hematopoietic system. Affected patients experience severe bleedings, autoimmunity, immunodeficiency, and an increased risk of hematopoietic malignancies. Since several decades, patients with WAS have been treated successfully using allogeneic hematopoietic stem cell transplantation – this procedure may however be associated with significant morbidity as well as mortality, in particular in case of HLA-disparity. Hematopoietic stem cell gene therapy has been developed as an alternative, but oncogene activation secondary to insertional mutagenesis can lead to oncogenesis. More recent therapeutic approaches include the use of self-inactivating lentiviral vectors, promising a better safety-to-risk ratio. Therapeutic efficacy and safety are currently being assessed. New methods of genomic engineering employing zinc finger nucleases, TALENs, and CRISPR-Cas as tools may offer new perspectives to site-specific genomic repair of disease-causing mutations.


Acute Myeloid Leukemia Hematopoietic Stem Cell Transplantation Chronic Granulomatous Disease Actin Polymerization Preimplantation Genetic Diagnosis 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.


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Copyright information

© Springer International Publishing Switzerland 2016

Authors and Affiliations

  1. 1.Dr. von Hauner Children’s HospitalLudwig-Maximilians-Universtität MünchenMunichGermany
  2. 2.Koch Institute for Integrative Cancer ResearchMassachusetts Institute of Technology (MIT)CambridgeUSA

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