Clinical Challenges: Gene Therapy for Inherited Retinal Diseases
The FDA approval of UF’s AAV technology in voretigene neparvovec (Luxturna) for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy almost 4 years ago marked a turning point in gene therapy.
After Nerve-Wracking Eye Surgery, the World Comes into Focus for Early Gene Therapy Recipient (STAT)
(This is a story about the first recipient of Luxturna, which is the first gene therapy for a genetic disorder approved by the FDA. UF Professor William Hauswirth’s work with AAV, in conjunction with inventors from University of Pennsylvania and Cornell University, is the technology behind Luxturna, marketed by Spark Therapeutics.) The machine looked like […]