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.
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.
Topics in the Gene Therapy and Other Medical Advances chapter
- 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?
The information on this site should not be used as a substitute for professional medical care or advice. Contact a health care provider if you have questions about your health.