Wiskott-Aldrich syndrome: Long-term results of gene therapy developed

A single infusion of genetically corrected autologous hematopoietic stem cells has been shown to provide lasting clinical benefits for patients with Wiskott-Aldrich syndrome. This rare genetic disorder impairs immune and platelet function, which exposes affected children from an early age to severe infections, bleeding episodes, eczema, and autoimmune complications.

The gene therapy approach offers a durable solution to the health challenges associated with the condition.