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Three University of Pennsylvania researchers have been honored by The Sanford and Sue Greenberg Prize to End Blindness by 2020 for their research, which led to the first Food and Drug Administration-approved gene therapy for a genetic disease. Gustavo D. Aguirre of the School of Veterinary Medicine and Jean Bennett and Albert M. Maguire of the Perelman School of Medicine, together with William Hauswirth of the University of Florida, are recipients of the Outstanding Achievement Prize, awarded in a virtual ceremony.
The four scientists share a $1 million prize, funds that will go to support further laboratory and clinical research that advances vision science. Together, their work going from an animal model of disease to human clinical trials led to an FDA-approved gene therapy for Leber congenital amaurosis (LCA) caused by a mutation in the RPE65 gene, a retinal disease that causes visual impairments beginning in infancy. Now commercialized and used routinely, this treatme