A Cure for “Bubble Boy” Disease

Scientists have used gene therapy to cure babies with a rare disease brought into cultural consciousness by David Vetter, who became known as “bubble boy.”

The study focused on children with X-linked severe combined immunodeficiency (SCID-X1): the most common form of a condition where the interleukin-2 receptor subunit gamma (IL2RG) gene mutation stops the body from producing a protein needed for the immune system to function. This renders the body unable to fight infection.

Without treatment, most children with SCID-X1 die by their first birthday. Currently, patients diagnosed with the condition can be cured if they can find a viable bone marrow stem-cell donor.

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