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Gene Therapy Promising for Treating 'Bubble' Children

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Gene Therapy Promising for Treating 'Bubble' Children

Children's Health

Gene Therapy Promising for Treating 'Bubble' Children

Gene Therapy Promising for Treating 'Bubble' Children

  • Download
  • <iframe src="https://www.npr.org/player/embed/4245773/4245774" width="100%" height="290" frameborder="0" scrolling="no" title="NPR embedded audio player">

British scientists report they've corrected a genetic defect in four children born with no immune system. The finding helps confirm the success of a French group that treated Damian Rodriguez, a Louisiana boy recovering after gene therapy helped his immune system behave normally — but also, inadvertently, triggered leukemia. Hear NPR's Richard Knox.