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

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.

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Bibliographic Details
Published in:Rare Dis
Main Authors: Braun, Christian Joerg, Witzel, Maximilian, Paruzynski, Anna, Boztug, Kaan, von Kalle, Christof, Schmidt, Manfred, Klein, Christoph
Format: Artigo
Language:Inglês
Published: Taylor & Francis 2014
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Addendum
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4755244/
https://ncbi.nlm.nih.gov/pubmed/26942098
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4161/21675511.2014.947749
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