New gene therapy for 'bubble boy' disease appears effective, safe

A new form of gene therapy for boys with X-linked severe combined immunodeficiency syndrome , a life-threatening condition also known as "bubble boy" disease, appears to be both effective and safe, according to a collaborative research team from Dana-Farber/Boston Children's Cancer and Blood Disorders Center and other institutions conducting an international clinical trial. Early data suggest that the therapy may avoid the late-developing leukemia seen in a quarter of SCID-X1 patients in pioneering gene therapy trials in Europe more than a decade ago.

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