International gene therapy trial for 'bubble boy' disease shows promising early results
Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a fatal genetic immunodeficiency also known as "bubble boy" disease, who were treated as part of an international clinical study of a new form of gene therapy. The mechanism used to deliver the gene therapy is designed to prevent the serious complication of leukemia that arose a decade ago in a similar trial in Europe, when one-quarter of boys treated developed the blood cancer. Researchers from the Dana-Farber Cancer Institute presented the study results annual meeting of the American Society of Hematology, on behalf of the Transatlantic Gene Therapy Consortium.
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