What Diseases Is Gene Therapy Currently Being Tested On?

Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Researchers are still studying how and when to use gene therapy.

Currently, in the United States, gene therapy is available only as part of a clinical trial.

Has gene therapy been approved in the United States?

Gene therapy is currently available primarily in a research setting. The U.S. Food and Drug Administration (FDA) has approved only a limited number of gene therapy products for sale in the United States. You may wish to refer to a list of gene therapy clinical trials that are accepting (or will accept) participants.

When has gene therapy been used?

Twenty years ago, gene therapy was touted as a cure for everything from cancer to cystic fibrosis. Now it is finally starting to fulfil its promise. In 2012, Glybera became the first gene therapy to be approved, for people with a rare disorder that makes them unable to process dietary fat.

Is Gene Therapy safe to cure genetic disorder?

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective.

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