David Vetter, known as 'bubble boy,' who died in 1984, is perhaps the most famous sufferer of severe combined immunodeficiency.
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.”
Scientists at St. Jude Children's Research Hospital recruited eight children from an existing clinical trial of patients with the disease, and collected samples of their bone marrow. A virus was then used to transport a corrected version of the gene into the DNA of their blood stem cells. Study co-author Dr. Mort Cowan, a professor of pediatrics at University of California, San Francisco where four of the children were treated, commented: “While longer follow-up is needed to assess any late effects of treatment, these results suggest most patients treated with this gene therapy will develop a complete durable immune response without side effects.”
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