Gene therapy

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New gene therapy for 'bubble boy' disease appears effective, safe Gene Therapy Drawing, How Gene Therapy Works, Genetic Regulation Diagram, Immunoglobulin Gene Structure, Genetic Lifeform And Disk Operating System, Boston Childrens Hospital, Bubble Boy, Gene Therapy, Medical Illustration

A new form of gene therapy for boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), 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…

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