What gene therapy is used for hemophilia?
Updated: Oct 5
Gene therapy for hemophilia targets the genetic defect responsible for the body’s inability to produce clotting factors—factor VIII (for hemophilia A) or factor IX (for hemophilia B). The treatment introduces functional copies of the F8 or F9 genes into liver cells using viral vectors, such as AAV (adeno-associated virus). These functional genes enable the production of the missing clotting factors, significantly reducing or even eliminating the need for routine injections of clotting factor concentrates. A notable example is Roctavian (valoctocogene roxaparvovec), which has demonstrated remarkable success in lowering bleeding episodes for hemophilia A patients, offering a more sustainable treatment option.
Got more to add on this topic? Share your thoughts in the comments below!
Keep exploring with Gene Tech Times
Free guide: subscribe to the Gene Tech Times Briefing on our homepage and get Every FDA-Approved Gene Therapy at a Glance in your inbox.
Watch: 30-second gene therapy Shorts on Gene Tech Times Academy.
Read: The Cheat Codes of Our Genes, real stories of mutations that make some people immune to disease.
New to gene therapy? Start with The Life-Changing Power of Gene Therapy.
As an Amazon Associate, Gene Tech Times earns from qualifying purchases.
Comments