NPR

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.
Researchers used a gene-carrying virus to fix blood stem cells that were then used to treat patients with beta-thalassemia. Source: Power and Syred

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

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