Is gene therapy available to treat my disorder?
Gene therapy is largely limited to research settings, with only a handful of FDA‑approved treatments, while dozens of clinical trials are exploring its use for many conditions.
Current Availability
Gene therapy is currently available primarily in a research setting. The U.S. Food and Drug Administration (FDA) has approved only a small number of gene therapy products for sale in the United States. For example, FDA‑approved gene therapies are available for conditions that include a rare eye disorder called Leber congenital amaurosis, a form of skin cancer known as melanoma, and a genetic muscle condition called spinal muscular atrophy. Other genetic therapies have been approved for blood‑cell cancers such as lymphoma and multiple myeloma. Gene therapies to treat additional conditions have been approved in other countries.
Clinical Trials
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.
Finding Trials
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 or gene or genome editing clinical trials that are accepting (or will accept) participants.
An explanation of the clinical trial process and how to find a clinical trial is presented by the American Society of Gene and Cell Therapy.
Related Topics
- What is gene therapy?
- How does gene therapy work?
- Is gene therapy safe?
- What are the ethical issues surrounding gene therapy?
- Is gene therapy available to treat my disorder?
- What are CAR T cell therapy, RNA therapy, and other genetic therapies?
- What are mRNA vaccines and how do they work?
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