Answers to Common Questions Asked about Gene Therapy for Hemophilia

Did you know?

Gene therapy is the use of genetic material to treat or prevent disease. Research into gene therapy for hemophilia has been underway since the 1990s.

The goal of hemophilia gene therapy is to get a working copy of a gene into the body so the body can then produce functional factor VIII or IX on its own.

Gene addition is the most successful and most practical type of gene therapy, which involves adding a new, functional gene. Most hemophilia gene therapy clinical trials involve gene addition.

These are answers to a few of the many questions people have about gene therapy. Want to learn more? Click on any of the sections below to learn more.

• Gene Therapy Defined
• Who is Eligible for Gene Therapy
• What I Can Expect from Gene Therapy
• Risks Associated with Gene Therapy
• Gene Therapy and Reproduction
• Gene Therapy Clinical Trials
• Gene Therapy and Inhibitors
• Psychosocial Issues and Gene Therapy
• General Information

Source: National Bleeding Disorders Foundation