Study Shows Feasibility of Using Gene Therapy to Treat Rare Immunodeficiency Syndrome
JAMA - Journal of the American Medical AssociationIn 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.