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 (WAS) is caused by loss-of-function mutations in the WAS gene. The condition is characterized by thrombocytopenia (low platelet count), eczema, and recurring infections. In the absence of definitive treatment, patients ...



