A clinical trial of gene therapy has been conducted by Genethon* in France and in Great Britain and treated 7 children suffering from the Wiskott Aldrich Syndrome. Preliminary results published this week in JAMA are extremely encouraging.
The genetic disease of the immune system called « Wiskott Aldrich Syndrome » is induced by the mutation of the WAS gene which, when functional, produces the WAS protein essential to the function of white blood cells and the production of blood platelets. In this disease, the WAS protein is not produced and the cells are no longer capable of fighting off microbes and of performing their duty as sentinels of the body. The children affected by this disease, mostly boys, contract multiple infections ; suffer from eczema, severe dermatitis and severe hemorrhages. They cannot live a “normal life”.