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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 (https://www.fda.gov/vaccines-blood-biologics/cellular-genetherapy- products/approved-cellular-and-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 (https:// 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 listof gene therapy clinical trials (https://clinicaltrials.gov/search?term=%22gene+therapy%22) that are accepting (or will accept) participants.
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