Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis.
نویسندگان
چکیده
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.
منابع مشابه
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ورودعنوان ژورنال:
- Rare diseases
دوره 2 1 شماره
صفحات -
تاریخ انتشار 2014