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Potential New Gene Therapy for Blinding Disease, Study Reports

The last year has seen milestones in the gene therapy field, with FDA approvals to treat cancer and an inherited blinding disorder. The researchers, from Penn's School of Veterinary Medicine and Perelman School of Medicine, in collaboration with University of Florida scientists, developed a therapy that effectively eliminates the abnormal copy of rhodopsin, a light-sensing molecule, and then restores it with a healthy copy of the protein??