Answers to Common Questions Asked about Gene Therapy for Hemophilia

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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.

Source: National Bleeding Disorders Foundation

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