Gene therapy shows early success against 'Bubble Boy' disease
Thursday, April 21, 2016 - 12:00
in Health & Medicine
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.