Parent holds toddler close to their chest in loving embrace as child's hair blows in the wind

Seek. Learn. Know.

This site offers information and resources for patients, families, caregivers, healthcare teams, and any others who are seeking to learn more about the basics of genes and gene therapies for genetic diseases.

Illustration of the double helix of DNA

Gene therapy may offer patients and families new hope in the treatment of life-threatening inherited diseases

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.

Learn more

A new era in medicine


US Food and Drug
(FDA) approvals
for gene therapies

through February 20231


Clinical trials
conducted or ongoing worldwide
as of February 20202


Years of
scientific research


1. Approved cellular and gene therapy products. US Food and Drug Administration. Updated December 16, 2022. Accessed February 21, 2023. 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.