Gene therapy shows early success against ‘Bubble Boy’ disease

Posted by on April 20, 2016 7:14 pm
Categories: health

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.

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