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Wiskott-Aldrich syndrome (WAS) is an X-linked disorder associated with abnormalities in the actin cytoskeleton of all hematopoietic lineages resulting in immunodeficiency and thrombocytopenia with small platelets. Affected boys have a bleeding tendency, eczema, recurrent opportunistic bacterial and viral infections, and autoimmune disorders and are at risk for developing lymphomas.
U.K. and French investigators now report outcomes of treatment with WAS-gene–corrected autologous hematopoietic stem cells (HSC) for seven severely affected children (ages, 0.8–15.5 years). Patient CD34 cells were purified and seeded on cell culture bags, stimulated for 24 hours, and then transduced twice with a modified lentiviral vector. The genetically modified…