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 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.

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