Gene Therapy Trial Success for Wiskott-Aldrich Syndrome
October 22, 2013 | Terry Sharrer
Wiskot-Aldrich Syndrome is a rare but fatal x-linked primary immunodeficiency that presents bleeding and blood cancers. To treat it, research physicians in Milan, Italy and Houston, Texas took bone marrow stem cells from three boys, “infected” those cells with an engineered virus (HIV backbone with a Wiskott-Aldrich protein gene inserted), and then reinfused the corrected cells. The advantage of using HIV was that it inserted itself into a chromosome at a location removed from the cancer promoting gene which had compromised earlier vectors. The patients responded immediately and have remained stable, without cancer or immunodeficiency, for as much as 30 months. MORE