Hemophiliacs seek solace in gene therapy

In what's being called a landmark study, researchers used gene therapy to successfully treat six patients with severe hemophilia, a blood-clotting disorder.

The study was preliminary and involved only six patients. Other promising early attempts to use gene therapy against hemophilia ultimately failed. But a single infusion using the new treatment worked in some patients for more than a year, boosting their clotting ability significantly.

"I think this is a terrific advance for the field. It's a good lesson in terms of don't give up on good ideas," says Dr Ronald Crystal, chairman of genetic medicine at New York City's Weill Cornell Medical College.