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.

Keywords:

• Wiskott-Aldrich-Syndrome
• gene therapy
• insertional mutagenesis
• leukemia
• immunodeficiency

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Disclosure of Potential Conflicts of Interest

No potential conflict of interest was disclosed.