Gene therapies are being developed to treat many types of genetic diseases, including those that affect the neuromuscular system, central nervous system, liver, eyes, and other tissues. Gene therapy can target specific genes that lead to disease by helping to replace, bypass, or silence the gene that isn't working properly.
How genes work
Know the basics of genetics to help you understand the role genes play in inherited diseases.
How gene therapy works
Learn how gene therapy works to regulate the way a gene functions, so the cell can produce better functioning proteins
How gene therapy is used
See how gene therapies are being used to treat rare inherited diseases
Find additional resources and information about organizations offering support.
Be equipped with questions to ask your doctor about clinical trials in gene therapy.
US Food and Drug
for gene therapies
through February 20231
conducted or ongoing worldwide
as of February 20202
1. Approved cellular and gene therapy products. US Food and Drug Administration. Updated December 16, 2022. Accessed February 21, 2023. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products 2. Maldonado R, Jalil S, Wartiovaara K. J Community Genet. 2021;12:267-276. 3. Asher DR, et al. Clinical development on the frontier: Gene therapy for Duchenne muscular dystrophy. Expert Opin Biol Ther. 2020;20:263-274.