Patients with the most severe form of the immune condition Wiskott Aldrich Syndrome have been successfully treated using gene therapy at GOSH.
The treatment meant that the children went from spending an average of 25 days in hospital in the two years prior to gene therapy to no days in the hospital in the two years after the treatment. It also allowed for one child who was confined to a wheelchair to return to normal physical activities without the use of the chair.
Wiskott Aldrich Syndrome (WAS) is a genetic condition that affects between one and 10 children in every million worldwide and reduces their ability to fight infection. Symptoms can include bleeding episodes, eczema and other recurrent skin infections, and autoimmune disease although there is a broad spectrum of severity within the disease with some children being more affected than others. The most severely affected children often need to spend time in hospital.
The condition can very successfully be treated by giving children a bone marrow transplant where faulty immune cells are replaced by working donor cells, although this relies on donors being a good match for patients. Without transplantation, patients with WAS often don