New gene therapy success in a rare disease of the immune system

The efficacy of gene therapy treatment for Wiskott-Aldrich syndrome has been demonstrated by researchers. Wiskott-Aldrich syndrome is a rare congenital immune and platelet deficiency which is X-linked and has an estimated prevalence of 1/250,000. It is caused by mutations in the gene encoding the WAS protein (WASp) expressed in hematopoietic cells. This disease, which primarily affects boys, causes bleeding, severe and recurrent infections, severe eczema and in some patients autoimmune reactions and the development of cancer.

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