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Gene Therapy

Physicians and researchers affiliated with the UF Genetics Institute are world leaders in the safe transfer of corrective genes as a therapy for genetic diseases. It was UF geneticists who discovered the unique lifecycle of the harmless virus called AAV and advanced its use as a carrier for therapeutic genes in the treatment of diseases such as cystic fibrosis, congenital Leber’s amaurosis, an incurable form of blindness, and Pompe disease, a genetic disease characterized by heart failure and muscular dystrophy.