News & AnalysisClinical Research

Gene Therapists Celebrate a Decade of Progress

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Science  07 Oct 2011:
Vol. 334, Issue 6052, pp. 29
DOI: 10.1126/science.334.6052.29

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Summary

It has taken many years, but researchers may have reached a prized goal in gene therapy: lowering the risk of uncontrolled bleeding in patients with hemophilia. At a meeting last week, researchers reported that six patients who received a virus engineered to carry a gene for a blood-clotting protein called factor IX needed fewer transfusions of the protein for as long as 18 months; some didn't require any transfusions. One patient developed an immune response to the viral vector, but this side effect was successfully treated with drugs. Some researchers say these results mark a watershed for the long-struggling field.