जेनेटिक सिंड्रोम और जीन थेरेपी जर्नल
खुला एक्सेस

अमूर्त

The Need for Gene Therapy for the Effective Treatment of Hemophilia

Tung Wynn, William Slayton and Roland Herzog

Gene therapy is promising to patients with hemophilia, with or without inhibitors. It offers the promise of life without the risk of bleeding after a simple injury and chronic arthropathy. It provides the promise of life without IV infusions for factor replacement. It makes the promise that no person with hemophilia will have to live a tragic life. Restoring a patient’s ability to make a protein that was congenitally deficient and all but curing the disorder is the goal of everyone treating any heritable disease and is the goal of gene therapy for hemophilia patients. Ultimately, gene therapy promises life without hemophilia. The goal and promise of gene therapy is well worth striving for.