Wiskott-Aldrich syndrome is a hereditary immunodeficiency disorder characterized by abnormal functioning of immune system and decreased ability to form blood clots. The abnormality in the platelets leads to easy bruising effects in case of prolonged bleeding.
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The white blood cells become non-functional and cause other immunological disorders in Wiskott-Aldrich syndrome. It has been discovered that this disease is caused by the mutation in WAS gene (Xp11.4-p11.21) that instruct to make Wiskott-Aldrich syndrome protein.
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Some of the symptoms observed in the patients are thrombocytopenia, hematologic abnormalities, eczema, and malignancies. Genethon Inc. is in the process of developing autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene as a gene therapy for the treatment of Wiskott-Aldrich syndrome. Further, the University of Pittsburgh and Orchard Therapeutics Limited are also involved in the pipeline for Wiskott-Aldrich syndrome.
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