Gene therapy pioneered at St. Jude shows early success against “Bubble Boy” disease

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Adolescents and young adults with a severe inherited immunodeficiency disorder improved following treatment with novel gene therapy developed at St. Jude Children’s Research Hospital and at the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health. The results of this study appear today in the journal Science Translational Medicine.

The study involved five males with X-linked severe combined immunodeficiency disease (SCID-X1), also known as “Bubble Boy” disease, who were all treated at NIAID. 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.

About Sabina Covo

Sabina started her career as a journalist in 1999. She graduated with a Communications Degree from Florida International University and has worked as a journalist, TV and Radio Anchor and Reporter for different stations and print publications in Latin America and the US including Caracol, W Radio, Austrian National Television, America Teve, Canal sur, NBC and Telemundo. She served as the director of Media Relations for Gordon Reyes and Associates in Miami and was nominated for a Raising Star Award by the Coral Gables Chamber of Commerce. She has a Building our Community award for her collaboration with the March of Dimes, and is part of Sharing for Kids. Sabina is a columnist for El Nuevo Herald in Miami, and a journalist and TV personality for America Teve.

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