Doctors at St. Jude accepted an award from the Smithsonian this week for finding a potential cure for severe combined immunodeficiency (SCID), or 'bubble boy' disease.
It’s not often that a rare genetic immunodeficiency disorder captures the attention of the general public, but with an iconic John Travolta-led feature film centered on “bubble boy” disease, it seems the condition — formally known as severe combined immunodeficiency — is an exception.
Early treatment sometimes involved putting individuals in NASA-level protective suits to insulate them from germs. But the method made it difficult to live a normal life. In recent years, researchers have used bone marrow transplants to treat the condition, but the procedure requires a sibling who is a match, which many do not have.
The method, as Ewelina Mamcarz, MD, one of the St. Jude researchers involved in the project tells Yahoo Lifestyle, involves taking a deactivated HIV virus and using it to deliver an unmutated gene. “Dr. Sorrentino always said ‘What are viruses good at? They’re good at infecting cells,’” Mamcarz says of the doctor, who passed away from lung cancer last year. “So he used the property of that virus to carry that normal health gene into the cell.
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