Discovery In “Bubble Boy” Disease Gene Therapy

Scientists at St. Jude Children’s Research Hospital have developed a mouse model of a severe disease of the immune system that helps explain why gene therapy used to treat children with this disease at an institution in Europe caused some of them to develop leukemia. The disease, called X-linked severe combined immunodeficiency (XSCID), is caused by a mutation in a gene called gamma C that prevents the immune system from forming B and T lymphocytes. B lymphocytes produce antibodies, and T lymphocytes perform a variety of tasks such as helping B cells and killing cells that are infected with viruses.