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.
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