Gene therapy trial shows promise for curing a type of hemophilia

Good News Notes:

“A new gene therapy appears to serve as a functional cure for the most common type of hemophilia, early clinical trial results indicate.

Patients who received the one-time intravenous therapy continue to have a more than 90 percent decrease in bleeding events two to three years after their initial treatment, researchers reported Jan. 1 in the New England Journal of Medicine.

The therapy fixes a broken gene in liver cells that causes production of flawed factor VIII, a protein that plays a key role in blood clotting.

People with this genetic mutation have hemophilia A, the most common type of this bleeding disorder. Hemophilia A accounts for 8 out of 10 cases of hemophilia, researchers said.”

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