A Gene Therapy Breakthrough Could Offer a Treatment for the Rare and Deadly ‘Bubble Boy’ Disease

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Researchers used an experimental gene therapy to develop a possible treatment for a rare and deadly immune disorder known as "bubble boy" disease, the National Institutes of Health (NIH) announced Wednesday. Because of a gene mutation, babies who are born with X-linked severe combined immunodeficiency (X-SCID) do not develop immune cells properly, leaving them highly susceptible to infections.

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