Gene therapy shows early success against ‘Bubble Boy’ disease

Early results suggest novel gene therapy is safe and effective for treatment of inherited immune disease, according to scientists. The study involved five males with X-linked severe combined immunodeficiency disease (SCID-X1), also known as “Bubble Boy” disease. This inherited disorder involves a mutation in the IL2RG gene that affects males and occurs in 1 of every 50,000 to 100,000 live births, leaving them with little to no immune protection.