Is gene therapy available to treat my disorder?
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.
Hundreds of research studies (clinical trials) are under way to test gene therapy as a treatment for genetic conditions, cancer, and HIV/AIDS. If you are interested in participating in a clinical trial, talk with your doctor or a genetics professional about how to participate.
You can also search for clinical trials online. ClinicalTrials.gov, a service of the National Institutes of Health, provides easy access to information about clinical trials. You can search for a specific clinical trial or browse by health condition or sponsor. You may wish to refer to a list of gene therapy clinical trials that are accepting (or will accept) participants.