Peter Barta/St. Jude Children’s Research Hospital

Gene therapy works for bubble boy disease

Researchers have safely used gene therapy to treat eight baby boys born with an immune disorder known as “bubble boy” disease, named after a patient who grew up in the 1970s in a plastic enclosure to avoid germs. In the study, published 17 April in The New England Journal of Medicine, researchers used a harmless virus to insert a missing gene called IL2RG into bone marrow cells taken from infants with X-linked severe combined immunodeficiency disorder (X-SCID). The first X-SCID gene therapy trial 2 decades ago at first appeared successful, but some patients later developed leukemia because the new gene activated a cancer gene. The Scientist reports that the new study used a safer virus as well as chemotherapy to make room for the repaired cells, a step that more effectively restored the infants’ immune systems.

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