Study shows feasibility of using gene therapy to treat rare immunodeficiency syndrome

In a small study that included seven children and teens with Wiskott-Aldrich syndrome, a rare immunodeficiency disorder, use of gene therapy resulted in clinical improvement in infectious complications, severe eczema, and symptoms of autoimmunity, according to a study in the April 21 issue of JAMA , a theme issue on child health. Wiskott-Aldrich syndrome is caused by loss-of-function mutations in the WAS gene.
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