Gene Therapy For Inherited Blood Disorder Reduced Transfusions
A small study finds promise for using gene therapy to treat patients with beta-thalassemia, a blood condition that can cause severe anemia. The experimental treatment is in early development.
Gene therapy is showing promise for treating one of the most common genetic disorders.
Results of a study published Wednesday show that 15 of 22 patients with beta-thalassemia who got gene therapy were able to stop or sharply reduce the regular blood transfusions they had needed to alleviate their life-threatening anemia. There were no serious side effects.
"We're extraordinarily excited about these early results," says , a professor
