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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