New gene therapy for 'bubble boy' disease appears to be safe, effective
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 an international clinical trial with sites in Boston, Cincinnati, Los Angeles, London and Paris. Early data published in the New England Journal of Medicine suggests that the therapy may avoid the late-developing leukemia seen in a quarter of SCID-X1 patients in previous gene-therapy trials in Europe that took place more than a decade ago.
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