New Gene Therapy That Can Treat Severe Combined Immunodeficiency

bubble boy disease

Some diseases called immunodeficiency diseases due to the fact that they cause total destruction of the immune system. While some diseases are critical than others ‘bubble boy’ disease is a very dangerous disease which can affect the life of the children who suffer from it. In this disease, the patient might need to live in a sterile environment their whole life, without being able to socially interact with people.

A recent study conducted by a team of scientists from the St. Jude Children’s Research Hospital, were able to develop a new way to actually cure the disease. This is a breakthrough in the medicine field as humans who suffered from this disease were rarely able to live through a marrow transplant which is hardly effective and very dangerous.

The team was able to develop a treatment for the disorder called X-linked severe combined immunodeficiency (SCID-X1). The study and the results were posted in The New England Journal Of Medicine, where the patients were able to produce T-cells, B-cells and NK-cells.

The study was led by Dr Ewelina Mamcarz, who is a doctor at the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy. He commented on his results saying, “These patients are toddlers now, who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives. This is a first for patients with SCID-X1.”