Gene Therapy Shows Promise against Blood-Clotting Disease
December 8, 2017
(ABC News) – Gene therapy has freed 10 men from nearly all symptoms of hemophilia for a year so far, in a study that fuels hopes that a one-time treatment can give long-lasting help and perhaps even cure the blood disease. Hemophilia almost always strikes males and is caused by lack of a gene that makes a protein needed for blood to clot. Small cuts or bruises can be life-threatening, and many people need treatments once or more a week to prevent serious bleeding. The therapy supplies the missing gene, using a virus that’s been modified so it won’t cause illness but ferries the DNA instructions to liver cells, which use them to make the clotting factor.