Gene Therapy Keeps “Bubble Boy” Disease at Bay in 8 Children

December 07, 2013

boy patient gene therapy trial

Researchers say they are achieving success in curing the genetic defect that causes some children to be born without immune defenses, a rare condition made famous in the 1970s by a Texas boy who lived most of his short life in a sterile "bubble,” NPR reports. Scientists now report that 8 out of 9 young children given gene therapy for a type of severe combined immunodeficiency disease, called SCID-X1, are alive and living amid the everyday microbial threats that would otherwise have killed them.

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