Gene therapy has reversed a dangerous immune disease in two babies, keeping them healthy for nearly a year so far. In a third child, treated 4 months ago, the therapy also appears to be working, researchers in France report. The babies no longer need to be kept in isolation.
The findings, described in the April 28 Science, come at a time when gene therapy is under fire in the United States. The Food and Drug Administration terminated one study in which a teenage boy died. Other gene-therapy trials deemed to pose health risks have been suspended, casting doubt on the technology's future in this country.
Although including only three patients and fighting a disease that's rare, the new study may put to rest the frequent criticism that gene therapy hasn't cured anyone, says Alan W. Flake, a pediatric surgeon at Children's Hospital in Philadelphia. "It's a landmark paper in that it's the first clear-cut, demonstrated treatment with gene therapy—of any disease