Gene therapy for blindness clears hurdle in mice
...ard developing a gene therapy to treat people with retinitis pigmentosa, an inherited disease that attacks the ...t to maintain vision in people who are affected by retinitis pigmentosa," Gorbatyuk said. "The second step, introducing the normal gene to the retina, will show ...UF researchers awaken vision cells in blind mice
...s that begin with the destruction of rods, such as retinitis pigmentosa, which affects about 1 in 3,000 Americans. "This is the first to my knowledge of a cone-targeted gene therapy that restores function in an animal model where cones are the primary defect," said Richard Weleber, Ph.D., a professor of mole......terventions for retinal degenerative diseases such retinitis pigmentosa. ...