News of the WeekGene Therapy

β-Thalassemia Treatment Succeeds, With a Caveat

See allHide authors and affiliations

Science  11 Dec 2009:
Vol. 326, Issue 5959, pp. 1468-1469
DOI: 10.1126/science.326.5959.1468-b

You are currently viewing the summary.

View Full Text

Log in to view the full text

Log in through your institution

Log in through your institution


Gene therapy researchers are approaching a key milestone: They appear to have controlled an inherited blood disorder called β-thalassemia that's more common than any disease treated so far with gene therapy. A young man who received new genes to repair blood cells no longer needs regular transfusions and, 2 years later, seems healthy. Describing this success last week to a U.S. review panel in Bethesda, Maryland, a researcher added a caveat: The inserted gene may have turned on growth signals, raising the potential for cancer.